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Your Voice Is Everything
When the Duchenne and Becker community speaks, the world listens — not just because of what you say, but because of how deeply it comes from the heart. In recent weeks, families across our community have continued to share their stories. Real life. Real emotion. Real impact. We know how hard that is. It takes…
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Elevidys shipments for ambulatory DMD patients to resume
Sarepta Therapeutics will soon resume shipping Elevidys (delandistrogene moxeparvovec-rokl) for ambulatory Duchenne muscular dystrophy (DMD) patients, following a review of safety data by the U.S. Food and Drug Administration (FDA), which recommended the voluntary pause on the gene therapy end. “Last week, at the suggestion of FDA, Sarepta made the difficult decision to pause shipments of…
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Sarepta to Resume ELEVIDYS Shipments to Ambulatory Patients After FDA Recommends Removal of Voluntary Hold for Ambulatory Population
The U.S. Food and Drug Administration (FDA) has announced that the agency is now recommending the removal of Sarepta Therapeutics’ voluntary hold for ambulatory patients eligible to receive ELEVIDYS. In its own press release, Sarepta reported that it will resume shipping ELEVIDYS to sites of care for treatment of ambulatory patients with Duchenne imminently. This…
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Simply Stated: Updates on Friedreich’s Ataxia (FRDA)
Friedreich’s ataxia (FRDA) is an inherited neuromuscular disease that primarily impacts the nervous system and heart and affects about one in 50,000 people worldwide. FRDA is characterized by a slow, progressive loss of limb coordination (ataxia) and effects on speech and swallowing. Multidisciplinary care can improve the quality of life of people living with FRDA…
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Del-zota for DMD awarded FDA breakthrough therapy designation
The U.S. Food and Drug Administration (FDA) granted breakthrough therapy designation to delpacibart zotadirsen, known as del-zota, as a treatment for people with Duchenne muscular dystrophy (DMD) amenable to exon 44 skipping. This designation is intended to speed the development and review of therapies for serious conditions when early clinical evidence is suggestive of a…
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How art can foster connection in the disability community
Last Sunday, my girlfriend, Amanda, and I co-led an art workshop that brought us both joy. The accessible art journaling session, “Why your story matters: Where art meets wellness,” was held at the Bishan Public Library in the northern part of Singapore and was part of the National Library Board’s “Body|Brain|Being” inclusive wellness program, which…
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Avidity Biosciences granted Breakthrough Therapy Designation for exon-44 skipping therapeutic, del-zota
CureDuchenne was an early investor in Avidity Biosciences in 2016, and congratulates the company for receiving Breakthrough Therapy Designation from the US FDA for delpacibart zotadirsen (abbreviated as del-zota), their exon 44-skipping agent for the treatment of Duchenne. Why is this important: The FDA grants Breakthrough Therapy Designations to speed up the development and review of drugs for serious…
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FDA Grants Breakthrough Therapy Designation to Avidity’s del-zota
Avidity Biosciences, Inc. today shared that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to del-zota (AOC 1044) for the treatment of Duchenne in individuals amenable to exon 44 skipping. Del-zota is currently being assessed in the Phase 2 EXPLORE44 Open-Label Extension (EXPLORE44-OLE) trial and is the first of multiple exon…
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Sarepta voluntarily halts Elevidys shipments in US after FDA request
Sarepta Therapeutics is voluntarily halting all shipments of Elevidys (delandistrogene moxeparvovec-rokl) in the U.S. after a request from the U.S. Food and Drug Administration (FDA). The company had initially refused to pause the shipments of its gene therapy for Duchenne muscular dystrophy (DMD) for individuals with DMD who are able to walk, but has since agreed.…
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The importance of advance directives, especially with MD
Amid the hustle and bustle of daily life, many people tend to put off what I like to call “after-life” planning. It sounds a bit less scary than “end-of-life.” When I was diagnosed with muscular dystrophy at the age of 12, long-term planning never crossed my mind. (Do any kids think more than five minutes…
