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Avidity’s New Access Program Marks a Milestone for the Duchenne Community
In 2018, CureDuchenne was the only Duchenne-focused organization to invest in Avidity Biosciences, recognizing the potential of their novel RNA-targeting approach for individuals with Duchenne muscular dystrophy long before it reached clinical trials. Today, we’re proud to celebrate a milestone that reflects exactly how patient-focused progress can work when scientific innovators, regulatory agencies, and a dedicated community come together with a shared…
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Finding meaning at 30 through my disease communities
Recently, my role at Shalom Medcare shifted from marketing communications assistant to brand executive. This move aligns my professional work with my long-standing identity as a disability and patient advocate. Shalom Medcare is a Singapore-based medical escort and transport provider, and the new role strengthens the connection between my lived experience as a Duchenne muscular…
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Avidity Biosciences Announces U.S. Managed Access Program for del-zota
Avidity Biosciences, Inc. has announced its Managed Access Program (MAP) for delpacibart zotadirsen (del-zota) for eligible individuals living with Duchenne who are amenable to exon 44 skipping in the United States. MAPs allow a limited number of patients to receive an investigational therapy that is still under U.S. Food and Drug Administration (FDA) review outside…
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Avidity announces a Managed Access Program for del-zota, for eligible individuals with Duchenne amenable to skipping exon 44
As an early funder of Avidity Biosciences, CureDuchenne is so happy to share that Avidity has launched a Managed Access Program (MAP) for its investigational therapy del-zota (delpacibart zotadirsen). Under an FDA-authorized treatment protocol, individuals with Duchenne who are amenable to skipping exon 44 will be able to be treated via participating healthcare providers. Enrollment…
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Clinical Research Alert: Real World Study of Male Fertility Following Risdiplam Treatment
Researchers at Genentech Inc. are seeking adult males treated with risdiplam (Evrysdi) for spinal muscular atrophy (SMA) to participate in an observational study (MARLIN) to assess the effects of treatment on fertility. Risdiplam is FDA-approved for treating SMA in pediatric and adult patients. The current study aims to better understand the fertility experiences of men with…
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Quick Guide: Understanding Programs and Benefits for People with Disabilities
Along with the daily challenges of living with a disability, many individuals face the added weight of financial strain. Jody Ellis “Not only do people living with disabilities face higher rates of unemployment and lower wages, but the cost of living with a disability is also higher, requiring about 29% more income than a household…
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Becker Muscular Dystrophy Series – Part 2: Cardiopulmonary Care
The second session in PPMD’s Becker Muscular Dystrophy webinar series focused on cardiopulmonary care. Our clinical experts reviewed what every person living with Becker and their families should know about care for the heart and lungs. Speakers: Dr. Andreas Barth – Medical Director, Center for Inherited Heart Diseases; Associate Professor of Clinical Medicine and Genetic…
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Tis the Season for Holiday Hacks and Tips
For some, the holiday season means the hustle and bustle of hosting, traveling, gift giving, and gatherings. For others, the end of the year is a time to slow down, cozy up, and practice gratitude. Whether you are hosting and traveling this year or spending quiet time reflecting, we have everything you need for the…
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FDA approves new safety warning and updated prescribing information for Sarepta’s Elevidys
Letter to the community added November 17, 2025 The FDA has approved the new labeling information Sarepta provided for Elevidys, Sarepta’s gene therapy for Duchenne. This update comes after reports of fatal acute liver failure in non-ambulatory patients, and a review of all of the available safety data. The updates include: A Boxed Warning for…
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Insmed’s ASCEND Study and INS1201, an Investigational Gene Therapy for Duchenne Muscular Dystrophy
Watch HERE The post Insmed’s ASCEND Study and INS1201, an Investigational Gene Therapy for Duchenne Muscular Dystrophy appeared first on CureDuchenne.
