❝ I am excited to be a part of the MENA Congress for Rare Diseases 2025. This congress is an excellent platform to foster collaboration and knowledge exchange across researchers, clinicians, and individuals with rare diseases and their families to advance therapies for individuals with rare diseases. ❞

❝ I am delighted to share that I have been granted the privilege to speak at the MENA Congress for Rare Diseases 2025. This significant event is a remarkable opportunity for experts, researchers, and healthcare professionals from across the region to come together and discuss the latest advancements and challenges in the field of rare diseases. The congress is a real chance to foster collaboration, share knowledge, and promote innovative solutions to improve the lives of patients affected by rare diseases. Through a series of presentations and panel discussions, the event will provide a dynamic platform for attendees to exchange ideas and insights. I strongly encourage all delegates to participate actively and contribute to our collective effort in advancing rare disease research and treatment. Your engagement and inputs will be invaluable in making this congress a success. ❞

❝ It is an honor to be a speaker at the MENA Congress for Rare Diseases, one of the most significant scientific events in the field. I am privileged to return to this event to showcase how the biomarkers for Huntington disease (HD), awarded at the previous edition of this Congress, have contributed to the development of a promising therapy for HD. This year marks 20 years of research into the therapeutic applications of dental pulp stem cells for HD (NestaCell® product). In celebration of these two decades of dedication, I have the exceptional opportunity to share in vitro, preclinical, and clinical evidence that highlight NestaCell® as a promising therapy for HD. I hope this journey inspires the scientific community and stakeholders, fostering a collaborative environment to advance the development of a global network that drives research, development, and innovation in therapeutic strategies for rare diseases. ❞

❝ Join me at the MENA Summit for Autism and Neurodivergence, an exciting part of the MENA Congress for Rare Diseases 2025! This event is a unique opportunity to contribute to the advancement of our region. I am looking forward to this opportunity to chair a session on discussing various ways to foster understanding of autism from a neuro-affirmative lens, share cutting-edge insights and innovative practices, and advocate for inclusive policies that enhance the lives of autistic individuals and families across the MENA region. As a neurodivergent professional, I am incredibly passionate about cultivating a neuro inclusive culture because inclusion is not a privilege but a right connected to the fundamental human value of belonging. ❞

❝ I am excited and proud to participate as a speaker and member of the scientific committee in this distinguished conference, the MENA Congress for Rare Diseases 2025. This annual conference plays a distinctive and key role in disseminating advanced knowledge at all levels for doctors, scientists, healthcare professionals, caregivers, parents, and patients. This extraordinary event brings together unique scientists from around the world, presenting their outstanding research on the diagnosis and treatment of rare diseases. It also involves several organizations that support rare diseases and families who contribute by sharing their experiences regarding their children’s illnesses. The primary goal of this unique meeting is to promote health and improve the quality of life for patients suffering from rare diseases. Moreover, this conference encourages and motivates young researchers to present their updated work, compete for recognition, and become an integral part of this exceptional congress for the MENA region. ❞

❝ أنا نشمية عبدالوهاب الفيلي والدة مزنة المصابة بمتلازمة الكورنيليا دي لانج، عضوة في جمعية الامارات للأمراض النادرة وإدارية في مجموعة أمهات العرب لأبطال كورنيليا دي لانج، وإدارية في مجموعة دعم أهالي أصحاب الهمم، أتقدم بخالص الشكر والامتنان لمؤتمر الأمراض النادرة في الشرق الأوسط وشمال افريقيا على تفانيه لرفع الوعي من خلال قصص تجاربنا وصمودنا كأمهات، لنجعل هذا المؤتمر منارة للتعاون والتعاطف والتقدم مع العوائل والأطباء والباحثين. لننقل التحديات في المجتمع والنظام الصحي الى السرعة في التنفيذ وبجودة عالية ❞

❝ Participating in the annual MENA Congress of Rare Diseases provides an invaluable experience to engage with leading experts and stakeholders to share insights and discuss breakthroughs in this field. The inclusion of a dedicated genetic counseling conference is particularly vital as it addresses the growing need for specialized support in understanding and managing the genetic aspects of rare diseases, ultimately improving outcomes and quality of life in the MENA region. ❞

❝ I am truly honored to represent the Foundation for Angelman Syndrome Therapeutics (FAST) at the MENA Congress for Rare Diseases, alongside Amelia Beatty, a dedicated member of FAST's Board of Directors. Together, we are excited to share FAST mission to drive transformative research and development programs forward as quickly as possible for everyone living with Angelman syndrome—regardless of age or genotype. At the congress, we will provide an update on the roadmap to a cure and highlight the latest research advancements. Our aim is to empower families in the MENA region with the knowledge and resources they need to make informed decisions for their loved ones. We look forward to connecting with you and advancing our shared goal of bringing meaningful change to the Angelman syndrome community. ❞

❝ It is an honor to present at the MENA Congress for Rare Diseases, a platform that has inspired countless professionals over the years. I am thrilled to introduce “Unified Healthcare Training: Empowering Medical and Patient Interfacing Professionals to Provide Compassionate Healthcare to People of Determination,” where we will explore the role of Applied Behavior Analysis (ABA) strategies in fostering inclusive healthcare practices. By integrating ABA principles, professionals can enhance their ability to support People of Determination, helping to create a healthcare system that is empathetic, equitable, and inclusive. This session provides practical tools and insights from behavioral experts, empowering attendees to drive meaningful change within their organizations. We invite you to join this vital conversation about compassionate care. Together, we can build a healthcare environment that truly values diversity, accessibility, and inclusion. ❞

❝ Progress in medical genetics is advancing rapidly. As we gain a deeper understanding of new genes and their role in disease mechanisms, we are also uncovering more rare diseases and their mutagenicity. Focused research is essential to classify the mutations associated with rare and undiagnosed genetic disorders. The MENA Congress for Rare Diseases presents a valuable opportunity to collaborate with leading international experts in the field, exchange knowledge, and explore potential management options to support patients and their families. ❞

❝ It is truly an honor to be part of the MENA Summit for Hemoglobinopathy, where we come together to share insights, advancements, and strategies for addressing hemoglobin disorders. With my background in hematology, I look forward to engaging in meaningful discussions that drive innovation and enhance patient care. I look forward to an insightful session with all of you. ❞

❝ I firmly believe that working in the biotechnology and pharmaceutical sector is a privilege, providing us with the opportunity to make a meaningful difference in the lives of patients and caregivers. When driven by purpose, hard work becomes a vocation rather than just a job. I am honored and look forward to sharing the innovative work my colleagues and I at Wave Life Sciences are doing every day in this critical field. The Middle East and North Africa (MENA) Congress for Rare Diseases is a vital forum for exchanging knowledge, learning from colleagues across the region and beyond, and advancing efforts to address rare diseases and unmet medical needs. ❞

❝ I am honored and excited to be part of the MENA Congress for Rare Diseases, a vital platform for advancing research, collaboration, and patient-centered care in the field of rare diseases. This congress unites experts, clinicians, and researchers committed to uncovering new insights and developing innovative solutions—a mission that deeply aligns with my passion for genomics research. Being part of this event is a privilege, as it provides an opportunity to share knowledge, exchange ideas, and contribute to meaningful discussions that drive real impact. I look forward to engaging with fellow experts, gaining new perspectives, and working toward improving outcomes for individuals affected by rare diseases. ❞

❝ Professional knowledge and willingness to share experiences make this congress truly valuable and productive. As a doctor, it is a great honor for me to present the results of the scientific work of a large team together with such enthusiastic and dedicated specialists. The topics for discussion inspire further development and the search for new solutions in medicine. Thank MENA Congress for Rare Diseases for openness, willingness to engage in dialogue, and commitment to making the world of health a better place. I hope that this congress will become not only a platform for the exchange of experience, but also a source of new ideas and inspiration. ❞

❝ I am honored to speak at the 2nd MENA Summit for Huntington Disease on "Approach to Chorea: Steps for Diagnosis". Chorea, a hallmark symptom of Huntington disease (HD), poses significant diagnostic challenges due to its resemblance to other treatable conditions such as Wilson’s disease, Sydenham’s chorea, and drug-induced movement disorders. My talk will focus on a practical, stepwise approach for neurologists, physicians, and pediatricians to improve diagnostic accuracy and patient outcomes. I will highlight the importance of differentiating HD from mimickers through careful clinical evaluation, targeted investigations, appropriate genetic testing, and advanced neuroimaging tools. By addressing these challenges, we can ensure timely diagnosis and tailored treatments for chorea, ultimately improving the lives of patients and their families in the MENA region and beyond. ❞

❝ As a parent of two wonderful children with neurodivergent traits, I have had the privilege of learning and growing through the unique experiences and challenges that come with neurodiversity. Embracing a strengths-based approach has been essential in supporting and nurturing their individual strengths and talents. I am truly honored to have been given the opportunity to speak at the MENA Congress for Rare Diseases 2025. It is my aspiration to impart my knowledge and insights to the audience, drawing from my own experiences. Furthermore, I am immensely grateful for the inclusive approach taken by the MENA Congress for Rare Diseases 2025, a unique event in the region, in recognizing the importance of neurodiversity. The inclusion of the MENA Summit for Autism and Neurodivergence as part of the MENA Congress for Rare Diseases 2025 is a significant step forward in creating a space where individuals and families affected by neurodivergent conditions can come together, share knowledge and foster understanding and acceptance within the community. ❞

❝ As we join the MENA Congress for Rare Diseases 2025, we are honored to learn from and collaborate with world-renowned experts distinguished by their exceptional contributions to genetic research. With their impressive history of innovation and discovery in the field of genetics, our partnership is poised to make significant contributions to the dialogue on rare diseases. We look forward to sharing our latest research, gaining insights from fellow experts, and working toward impactful, meaningful solutions for patients across the MENA region and beyond. ❞

❝ It is my pleasure to be among this distinguished group of experts, where I will update the audience on the progress made in sickle cell disease treatment. A better understanding of the pathophysiology of sickle cell disease has created opportunities for drug development to ameliorate the manifestations of this disease, giving hope to millions of patients around the world. ❞

❝ Rare diseases are a leading cause of infant mortality and lifelong disability, making them a global public health priority. Improving outcomes requires timely diagnosis and effective treatments, while also addressing clinical challenges. The 4th edition of the MENA Congress for Rare Diseases will undoubtedly increase awareness of rare diseases and enhance their visibility within healthcare systems. I am therefore thrilled to share our Belgian experience with BabyDetect, a tool developed to screen for 165 treatable pathologies from a drop of blood using NGS. Our results demonstrate the feasibility of mid-scale targeted genomic NBS, emphasize the value of integrating both genomic and biochemical techniques in NBS programs, and highlight some of the challenges this approach faces. I look forward to engaging with participants across multiple disciplines, exchanging valuable insights that drive research, with the common goal of achieving a better understanding of rare diseases, developing therapeutic and screening strategies, and ultimately improving patient care. ❞

❝ It is an honor to chair the 3rd MENA Rare Disease Symposium for Patients and Families at the MENA Congress for Rare Diseases 2025. This platform provides a vital space for collaboration, awareness, and advocacy, ensuring that patients and families feel supported and empowered. Through meaningful discussions and shared experiences, we can drive positive change and enhance the quality of life for those affected by rare diseases. I look forward to engaging with experts, caregivers, and families in a collective effort to build a more inclusive and informed community. ❞

❝ I am both excited and grateful to be part of the MENA Congress for Rare Diseases 2025. The line-up of speakers is extraordinary, and I look forward to seeing how each contribution will enhance the collective impact of the meeting. This congress is sure to push scientific boundaries while advancing the care we provide to patients with rare diseases. As the Dean of a Medical School, I believe that correlating clinical observations with basic science is essential in developing novel treatment modalities for rare diseases. ❞

❝ It is immensely satisfying to see a congress of this magnitude dedicated to addressing questions about different aspects of managing diseases and conditions that are not commonly encountered. Input from highly experienced and specialized professionals provides an amazing opportunity to gain firsthand knowledge about these conditions. Even more importantly, hearing from patients and families offers a window into the unique strengths and challenges these conditions present to them. This is truly a unique and valuable resource for everyone committed to working in this field. ❞

❝ MENA Congress for Rare Diseases 2025 will provide a unique platform for innovative discoveries and insightful discussions and collaborative networking for rare diseases. Our congress allows exchange of experiences and knowledge between all participants from the region and all over the world ❞

❝ Honored and excited to be part of the MENA Congress for Rare Diseases in the UAE. Having the opportunity to share the experience I faced, covering the many stages of identifying that my child was harboring an unexpected, unclear, and surprising condition, to how it was managed, understood, and then treated throughout the journey of discovery, acceptance, impact, and treatment. I believe this Congress is an amazing place to gather and bring together many forms of insights, both through professional and personal experiences. The desire to share and learn is one of the most important traits we each have to support one another, enabling a better and wider vision and awareness of how essential it is to stand together as a community for the healthy expansion of our global community, especially when it comes to individual health and well-being. Holding hands as individuals, patients, caregivers, parents, family members, friends, doctors, nurses, medical professionals of all kinds, pharmaceutical companies, healthcare companies, providers, hospitals, educational institutions, Congress organizers, etc., is key to making the world a better place for everyone, whatever their ‘condition’ may be. ❞

❝ I am honored to speak in the coming MENA Congress for Rare Diseases sharing Bahrain experience and addressing different achievements and challenges in the field of rare diseases. This exciting and interesting congress gives us the opportunity to present and discuss different topics including innovation in genetic testing and recent discoveries in rare diseases including new treatments. By hosting experts in the field of genetic and rare diseases from the region and all over the world, it will be an excellent platform for information sharing and recent updates exchanging. I am looking forward for this amazing congress. ❞

❝ In Middle East and North African countries (i.e., the MENA region), certain rare diseases are more common than in other areas of the world. Patients with such rare diseases require a high level of suspicion to diagnosis, may present to different subspecialists, and need a multidisciplinary approach. The MENA Congress for Rare Diseases is thus an important congress for practitioners in the MENA region that highlights relevant diseases which are often not well covered in other international conferences. The symposia of the congress and the broad range of specialties represented are impressive. I look forward to participating in and learning from the MENA Congress for Rare Diseases 2025. ❞

❝ I am deeply honored and grateful to have been invited to speak at the MENA Congress for Rare Diseases. This platform holds immense significance, bringing together incredible minds, advocates, and change-makers dedicated to improving the lives of individuals affected by rare diseases. As a parent navigating life with a child with special needs, the journey can be challenging, but it is also filled with lessons in resilience, hope, and the power of community. The Congress provided a space to share my story, connect with others who understand, and amplify the voices of families like mine. Together, we are fostering awareness, support, and positive change for those living with rare diseases. ❞

❝ I am honored to participate in the MENA Congress for Rare Diseases, bringing over 15 years of experience in pediatric biomedical research. My work focuses on developing innovative diagnostic methods for rare disorders, with a particular emphasis on lysosomal storage disorders and next-generation sequencing for genetic testing. As the former program director of the Austrian Newborn Screening, I led efforts to detect rare metabolic and endocrine disorders in infants. I also founded the Medical Laboratory ARCHIMEDlife to provide advanced diagnostic services for rare diseases. I look forward to sharing my research and collaborating with fellow experts to improve rare disease diagnostics and patient care. ❞

❝ I am honored to be invited as a speaker at the MENA Congress for Rare Diseases. With over 15 years of experience in the field of Applied Behavior Analysis (ABA), my work has focused on enhancing the quality of life for individuals with autism and developmental disabilities through evidence-based, compassionate strategies. This congress serves as a vital platform to share insights, collaborate with experts, and promote innovative approaches to supporting individuals with rare conditions and their families. Together, we can foster inclusion and create meaningful impacts across the region. ❞

❝ Improving care for rare disease patients requires global collaboration and continuous inspiration from various parts of the world. MENA Congress for Rare Diseases will be a wonderful opportunity to share experience in the field of Duchenne muscular dystrophy and to gain knowledge and learn about care approaches in different regions than I am used to working in. ❞

❝ It is my honor to participate in this esteemed gathering focused on rare diseases in the MENA region. The noble aim of this conference—to enhance the health and quality of life for individuals affected by rare diseases, particularly in our region—resonates deeply with my professional commitments. Conferences of this caliber are invaluable for fostering knowledge exchange and collaboration and play a crucial role in advancing our collective understanding and improving patient care. As a nutrition specialist and healthcare professional, I am dedicated to improving the lives of those grappling with metabolic disorders, including weight-related issues, alongside the unique challenges posed by rare diseases. This gathering provides a crucial platform for sharing expertise in dietary interventions and learning about the latest advancements in rare disease management. I extend my sincere gratitude to all involved in organizing and contributing to this event. Your efforts are vital in advancing our understanding and improving care for those with rare diseases in our MENA region. Together, we can make significant strides in enhancing the quality of life for these individuals and their families. ❞

❝ It is a great honor to be invited as a speaker at the esteemed MENA Congress for Rare Diseases 2025, and I am grateful for this opportunity. The future of rare diseases in the era of genomic medicine and artificial intelligence will be revolutionary. I have dedicated my work to rare diseases and am pleased to share my experience in this cutting-edge research, supported by evidence. By sharing these insights, I hope to contribute to the goal of early diagnosis and improved access to treatment, ultimately enhancing the quality of life for patients. ❞

❝ I am extremely honored to be invited as one of the speakers at the MENA Congress for Rare Diseases 2025. It is both a privilege and a responsibility to share my perspectives on Rett syndrome—our journey with our 8-year-old daughter, Johanna, a true Rett syndrome warrior. Through this platform, I hope to shed light on the challenges, triumphs, and unwavering resilience that define our experience, while also contributing to a broader conversation about awareness, advocacy, and support for individuals and families affected by rare diseases. ❞

❝ This is an incredible opportunity for a deep dive into learning more about rare diseases by listening to the latest advancements from experts. The MENA Congress for Rare Diseases 2025 gives me the chance to explore how media coverage can shape the narrative, promote acceptance, fight stigma, and focus on inspiring stories to create a better understanding of rare conditions. Once information is broken down and knowledge about rare diseases is shared, the door opens for empathy and inclusion. Looking forward to connecting, learning, and contributing so stronger bridges can be built between the research and medical community and the media. ❞

❝ Rare diseases present unique and significant challenges in diagnosis, treatment, and research due to their low prevalence and limited awareness. Many patients experience delayed or incorrect diagnoses, often waiting years before receiving proper medical attention. The rarity of these conditions also means that research funding and pharmaceutical interest are often limited, resulting in fewer treatment options. However, advancements in genomics, precision medicine, and international collaboration have greatly enhanced our understanding of rare diseases. To further drive research and improve access to care, increased awareness, patient advocacy, and policy support are essential. Additionally, greater funding and policy incentives, such as orphan drug designations, are crucial to improving outcomes for rare disease patients and accelerating the development of effective therapies. ❞

❝ It is a great pleasure to actively participate in this important conference on rare diseases, where I hope to expand my network of contacts and collaborators in the Middle East and North Africa region. This event is one of the most significant in the world in the field of rare diseases, where international collaboration plays a crucial role in translating advanced research into patient care. This translation is essential not only for improving diagnostic healthcare and shortening the diagnostic odyssey for many patients and their families but also for advancing genomic medicine, personalized treatments, and innovative therapies such as gene therapy and drug repurposing. I look forward to sharing my experience in translational research, from bench to clinical trials, with all attendees and to establishing meaningful scientific collaborations. ❞

❝ The MENA Congress for Rare Diseases is an incredible forum for advancing knowledge and establishing communities amongst clinicians, academic researchers, families, and industry partners. As an academic scientist, I am grateful for this conference for providing a window into the patient experience and clinical practice to inform ongoing therapeutic development. ❞

❝ I am truly honored to have the opportunity to share the genetic counseling experience from Oman at the upcoming MENA Congress for Rare Diseases 2025. I look forward to engaging with fellow experts and researchers as we explore the future of genetics and its impact on our world. ❞

❝ At this Congress, I look forward to engaging with fellow experts, exchanging insights, and collaborating on solutions that improve accessibility, reduce inefficiencies, and drive sustainable healthcare practices. Together, we can build a future where no patient is left waiting for essential treatment. ❞

❝ Inclusive workplaces are essential for the health and well-being of people with multiple sclerosis and other people of determination. Employment barriers extend beyond the workplace, impacting overall health and quality of life. From a public health perspective, creating accessible work environments helps reduce health disparities and promote long-term well-being. I look forward to contributing to the discussion on workplace inclusion as a public health priority, sharing recommendations and strategies that empower both employees and employers to foster healthier, more inclusive workplaces. ❞

❝ Participating in the MENA Congress for Rare Diseases 2024 was a truly inspiring experience, and I am deeply grateful to the organizers for creating such a meaningful event. It was an honor to share insights on stress management for parents of children with special needs and to engage with a community so dedicated to improving lives. The congress provided a unique platform for learning, collaboration, and the exchange of ideas, fostering a sense of unity and purpose among professionals, researchers, and advocates alike. I look forward to continuing this important work and am excited to share more insights at the coming congress, MENA Congress for Rare Diseases 2025. For anyone passionate about making a difference in the field of rare diseases, attending this congress is an invaluable opportunity to connect, contribute, and be part of a movement that truly matters. ❞

❝ Pushing scientific boundaries to improve the lives of rare disease patients requires collaboration across multiple disciplines. As members of this community, we strive to expand these boundaries by integrating insights from both coding and non-coding DNA using whole-genome data, combined with advancements in AI. I am excited to present our work at the MENA Congress for Rare Diseases, engage in meaningful discussions, receive valuable feedback, and contribute to this vibrant community to enhance patient outcomes. ❞

❝ أنا أم لطفلين يعانيان من مرض استقلابي نادر. فقدت طفلي الأول، واليوم يواجه طفلي الثاني نفس المرض، قررت أن أتحول من الألم إلى الفعل، انا و مجموعة من الأمهات الذين يعانون من الأمراض النادرة ندعم بعض من خلال نشر الوعي وتقديم الدعم النفسي وتنظيم فعاليات توعوية، وانه لشرف لي أن أتحدث في هذا المؤتمر عن تحديات الحياة مع الأمراض النادرة من خلال تعايشي مع مرض أطفالي، وأهمية مجموعات الدعم التي تسهم في تقبل الأسر لمرض أولادهم و دعم الأمهات نفسيا ❞

❝ Join me for a transformative experience at the MENA Congress for Rare Diseases! I’m thrilled to present “Nutritional Challenges in Managing Rare Diseases,” where we’ll explore how innovative nutrition strategies can significantly enhance patient care. It is your chance to be part of the conversation that will shape the future of rare disease management. Don’t miss this opportunity. Your insights and engagement are keys to making a real impact! ❞

❝ I’m delighted to be joining the MENA Congress for Rare Diseases to talk about the work we, as the patient advocacy community, have done to progress treatment options for the metabolic disorder, Galactosialidosis. Initially, we established The Galactosialidosis Network in the UK as a source of support and advocacy for individuals grappling with this ultra-rare inherited metabolic disorder. My interest in patient advocacy was born out of a deeply personal journey with my daughter, Clara, who battled this condition and sadly passed away on 6 March 2024. During our fight to save her life, our network gained global reach, fueled by the collaboration of devoted parents, clinicians, and our partnership with ISMRD—a champion for rare diseases worldwide—and Metabolic Support UK. It was challenging to garner interest from pharmaceutical companies for various reasons. Nevertheless, our mission swiftly expanded to embrace all children confronting Galactosialidosis. We were able to highlight the prevalence of rare diseases on a global scale, raising awareness and offering support to families in need. Our vision extended far beyond this, driving meaningful change by fostering collaboration between families, clinicians, and the scientific community. By leveraging my professional experience, we empowered the parent community to actively contribute to the pursuit of approved, enduring therapies for this rare disease. In less than a year, with a well-planned communication strategy and a focus on relationship-building, we successfully brought together all key stakeholders, including scientists and parents in this field. We equipped them with knowledge and advocated for groundbreaking scientific advancements. During this time, we earned the trust of both the scientific and patient communities, gaining the confidence to champion the needs of the rare disease community effectively. We are now very close to achieving an effective treatment after submitting a successful FDA application for the AAV8 therapy. This was made possible because we led and mobilized key individuals and organizations worldwide. It will be a great opportunity to share our journey at the MENA Congress for Rare Diseases and discuss the next steps needed to bring us even closer to our goal of initiating a clinical trial without further delay. ❞

❝ I had the privilege of participating in the MENA Congress on Rare Diseases previously, and it was an incredibly insightful and impactful experience. Conferences like this play a crucial role in strengthening the community and fostering collaboration among all stakeholders. This year, I am even more excited as the congress has expanded its scope to include the important topic of neurodiversity. Given that many rare diseases can coexist with neurodivergence, this inclusion is both timely and essential. I look forward to being part of this year’s event and continuing to learn, share, and grow alongside such a passionate and dedicated community ❞

❝ This conference is gaining strength every year. The amount of information, support, and new knowledge that are offered in this conference are immense. I had the privilege to speak and to present. I certainly look forward to learning at next year's congress ❞

❝ Having spoken at the conference last year, I consider it a privilege to return. I look forward to sharing what this journey with rare disease has taught me alongside other parents. ❞

❝ The MENA Congress for Rare Diseases is a unique scientific event in the region. I am honored to be a speaker and moderator at this congress. I will be sharing insights into the background of pharmacoeconomics and patient access to innovative treatments within the healthcare system in the UAE. ❞

❝ Balancing costs and benefits is essential in budget management and resource allocation for rare disease care. Pharmaco-economic evaluation enables the comparison of orphan drugs with supportive care, ensuring optimal use of resources while maximizing patient outcomes. The MENA Congress for Rare Diseases serves as an excellent platform to explore sustainable solutions for accessibility and affordability. I look forward to contributing to this vital discussion. ❞

❝ I am honored to participate in this important and highly scientific conference. Health economics now seeks to examine the factors that influence the industry’s costs and quality of care, especially in rare diseases. In response to the emerging national and international interest in rare diseases, it is crucial to connect, share diverse experiences across countries, and discuss the value of interventions and their impact on patients, healthcare systems, the economy, and society. This dialogue is essential for promoting health for all. ❞

❝ Throughout my university years, rare diseases have been a major area of interest. As a biotechnologist, I consider it my duty to study, understand, and contribute to the search for potential treatments for these conditions. Speaking at the MENA Congress for Rare Diseases 2025 will provide me with the opportunity to share my insights on Lesch-Nyhan syndrome, exchange knowledge with fellow experts, and broaden my perspective on other rare diseases. I encourage students, trainees, and colleagues to attend the Conference, as it will undoubtedly enhance their understanding of rare diseases. ❞

❝ I look forward to participating in the MENA Congress for Rare Diseases 2025. This is an excellent opportunity for stakeholders across the translational spectrum of targeted therapy development to share their expertise and to learn from professionals in related fields. ❞

❝ My name is Abdulla Lutfi, I am Autistic Savant, black and white Emirati artist. I am honored to be a part of the MENA Congress for Rare Diseases 2025 in partnership with Burjeel Medical City and honored to be speaking at the 1st MENA Summit for Autism and Neurodivergence. I see my autism not as a limitation but as a strength. It has given me a different way of seeing the world, and I realized I could use that to tell stories, not only about myself but about the people and places around me. I am proud to represent the UAE as an autistic savant artist, and I hope my work inspires others to embrace their differences and use their unique talents to share their voices. Every stroke of my pen is a reminder that we all have a special way of seeing and experiencing the world, and that should be celebrated. ❞

❝ I am excited and honored to participate in the MENA Congress for Rare Diseases. As a dedicated medical scientist in the global rare disease community, I have devoted most of my career to gaining valuable insights and serving patients with orphan diseases across North America and Europe. This international conference provides a unique opportunity to engage with esteemed colleagues from the Middle East and beyond. I look forward to sharing my knowledge, exchanging ideas, and learning from the diverse perspectives and expertise of those in attendance. This Congress will strengthen global collaboration efforts to enhance the understanding of rare diseases and improve patient outcomes worldwide. ❞

❝ I am glad to participate in the MENA Congress for Rare Diseases 2025. This congress serves as a platform to present the latest advancements, exchange cutting-edge scientific knowledge, and discuss the newest clinical applications aimed at improving the care of patients with rare diseases. ❞

❝ I am honored to participate in the MENA Congress for Rare Diseases, as it offers a valuable opportunity to enhance global awareness and share knowledge among healthcare professionals supporting children and families affected by rare conditions. As a neuromuscular nurse specialist, I am dedicated to advancing care quality and improving the lives of children living with neuromuscular disorders. ❞

❝ I am eagerly anticipating the opportunity to participate in the MENA Congress for Rare Diseases 2025, where I look forward to engaging with healthcare providers, researchers, advocates, and patients as we work collectively to ensure that no person living with a rare disease is left behind. This congress perfectly aligns with RDI’s commitment to deepening our engagement and support for people living with rare diseases (PLWRD) and their communities across the MENA region. The high quality of the presentations, the diversity of topics covered, and the strong international participation underscore the significance of this congress as a key platform for advancing our shared mission. At Rare Diseases International, our vision is to create a world where people living with rare diseases and their families experience a better quality of life through full recognition and comprehensive support. This congress is a vital step in achieving that vision by bringing together global stakeholders to collaborate, innovate, and advocate for those who are too often overlooked ❞

❝ It is my pleasure to be a part of the MENA Congress for Rare Diseases. This event provides a unique opportunity for the community, professionals, researchers, and government officials to come together and exchange ideas on improving the lives of those living with rare diseases. I am looking forward to speaking about the role of stakeholders in shaping inclusive frameworks and exploring the various ways to engage policymakers, the community, and industry stakeholders in driving social change. ❞

❝ As a dedicated adoptive mother of a child with a rare disease, I am honored to share my journey at the MENA Congress for Rare Diseases. My speech will focus on the intricate path of diagnosis and the transformative experience of motherhood when raising a child with special needs. I aim to shed light on the emotional and practical challenges faced during this journey, as well as the profound joys that come with it. Through my story, I hope to inspire and empower others navigating similar paths, fostering a deeper understanding and connection within our community. ❞

❝ I am honored to take part in the MENA Congress for Rare Diseases 2025, the largest event for rare diseases in the region. I am grateful to connect with patients and their families to exchange experience and knowledge. As a mother of two children with a rare disease, I am glad to get the opportunity to share my experience in the congress and raise the awareness about mitochondrial disorders. ❞

❝ Hailing from the eastern province of Saudi Arabia, where rare diseases are prevalent, I consider the MENA Congress for Rare Diseases a profoundly enlightening and enriching experience for all individuals contending with these health challenges. The MENA Congress for Rare Diseases serves as a pivotal regional summit concentrating on rare diseases and orphan drugs within the Middle East and North Africa (MENA) region. Its aim is to convene healthcare professionals, researchers, policymakers, and patient advocates to deliberate on advancements, exchange knowledge, and foster collaboration towards bettering the care and treatment of rare diseases. From a personal perspective, I look forward to delving into alpha thalassemia and assimilating the latest updates on treatment and advancements ❞