Cure Rare Disease

Spinocerebellar Ataxia Type 3 (SCA3) is a neuromuscular disease, affecting 1 to 5 in 100,000 people, and is the most common form of spinocerebellar ataxia. Mutations in the ATXN3 gene lead to toxic protein accumulation, impacting ataxin-3 enzyme function. Learn more about SCA3 and our efforts to develop a treatment for it using ASO technology in collaboration with Leiden University Medical Center: https://lnkd.in/gqFKkZp8 #SCA3 #RareDiseaseAwareness #CureRareDisease

We are excited to announce significant additions to our leadership team and Board of Directors. Markus Ward has joined us as Chief Advancement Officer, bringing a wealth of experience and passion to our mission. Additionally, we are excited to welcome Jeremy Sternberg, J.D., Klaus Veitinger, M.D., Ph.D., and Mike Williams to our Board of Directors. Their diverse expertise and commitment will undoubtedly strengthen our efforts in advancing our mission. Read More: https://loom.ly/Kq__Low #CureRareDisease

Introducing the Angel Treatment Pathway. Developed in collaboration with global value, access and policy expert, Eric Faulkner, and funded by the Muscular Dystrophy Association, the Angel Treatment Pathway is the first comprehensive approach mapped to-date in the United States that offers a potential avenue for ultra-rare disease patients to access potentially life-saving therapies. In addition to drafting the initial proposal for the Angel Treatment Pathway, our team held meetings with leading rare disease and advanced therapy associations, industry, payers and advocacy groups to gather feedback, insight, and pressure test the details of the proposal from various perspectives in the rare disease ecosystem. This assessment and roadmap shows that most of the components of the pathway can actionably leverage the practices and infrastructure of our existing health system. The Angel treatment pathway report also helps make a case for the value of shifting our thinking about rare disease. By the numbers, the collective of up to 8,000 rare diseases rivals or exceeds many of our most devastating diseases, but these patients often have poor or no options. By moving from a historical view of ultra-rare disease as thousands of small populations to a collective from a solutions perspective, we have the potential to advance cross-cutting solutions that can rise to meet this need. We are grateful to each of the collaborators who contributed valuable feedback for this initial draft, which we hope will be the beginning of solving the most pressing challenges to accessing potential therapies for ultra-rare disease patients. Visit our website to learn more and access the full report: https://loom.ly/IaRagS0 #RareDisease #AngelTreatmentPathway #CureRareDisease

In 2023, we raised a total of $3,759,840 from the generous support of our community. Over 92% of these funds have been allocated directly to research and development initiatives. This significant investment enables us to continue our work to advance potentially life-saving treatments for ultra-rare diseases. Thank you for your continued support in fueling our mission! Learn more in our 2023 annual report: https://loom.ly/mkXc4-U #CureRareDisease #Research #RareDisease

Join us in paving the way for a future where rare disease patients are no longer left behind. Together, we're advancing science and technology to make hope for safe and effective treatments a reality. Learn more: https://loom.ly/mkXc4-U #CureRareDisease #RareDisease #GeneTherapy

Register now to be part of the Disney Wine & Dine Half Marathon Weekend! Celebrating 15 years of excellence, this event promises four immersive days filled with iconic Disney Chefs, delectable food, and unforgettable running experiences. Join the CRD team today and prepare to embark on a journey of culinary delights and athletic achievement. Secure your spot now! https://loom.ly/FRdRXcY #DisneyWineAndDine

Establishing a cell line for Duchenne Muscular Dystrophy (DMD) is the first stage in our process to gauge efficacy of a potential therapeutic approach before moving into testing in mouse models. This crucial part of the research process involves several key steps and can take a considerable amount of time. Read our latest blog to learn more about this process and its importance in developing potential therapies for DMD. Read more: https://loom.ly/uV5ceE0 #DMD #RareDiseaseResearch #CRISPR #CureRareDisease

Our 2023 Annual Report is out! In this report, you will find a comprehensive overview of all our team accomplished last year and milestones for the years ahead, including updates on each of our drug development programs, technology, policy advancements, and much more. The work detailed here would not be possible without the commitment and dedication of each of our research collaborators, industry partners, board members, supporters, and staff. Thank you for your continued support of our work to transform realities for ultra-rare disease patients! View here: https://loom.ly/mkXc4-U #AnnualReport #RareDisease #CureRareDisease #CRD

Calling all Disney enthusiasts and avid runners! Join CRD's team at the Disney Wine & Dine Half Marathon Weekend, celebrating its crystal anniversary with four days at Walt Disney World® Resort. Learn more and register now! https://loom.ly/FRdRXcY #CRDTeam #DisneyWineAndDine #RunForACause

On March 16, we celebrated the grand opening of Cure Rare Disease's new laboratory in Woodbridge, Connecticut! Check out our latest blog for a recap and photos from the event. Thank you to our community and Senator Bluemthal for joining us to mark this milestone as we continue to drive research progress for ultra-rare diseases. Read now: https://lnkd.in/eWgS3Ksy #CureRareDisease #LabOpening #ResearchInnovation #NeuromuscularDiseases #RareDiseases