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MDA’s Guide to the Affordable Care Act (ACA)
For many people living with neuromuscular diseases, access to reliable health insurance can be the difference between receiving life-changing care and going without it. The Affordable Care Act (ACA), signed into law in 2010, created stronger protections for people in the United States living with disabilities, chronic illnesses, and preexisting conditions. Understanding the ACA can…
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An annual physical brings a sigh of relief to this Duchenne MD caregiver
I had my annual physical last week and when the doctor told me I was in good health, it was a relief. I let out a breath I hadn’t realized I was holding. I am a mother to seven: Lexi, 25; Max, 20; Chance, 19; Rowen, 17; Charlie, 15; Mary, 11; and Callie, 4. Max,…
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FDA Announces Advisory Committee Meeting to Review Capricor Therapeutics’ Biologics License Application for Deramiocel
The U.S. Food and Drug Administration (FDA) has scheduled an Advisory Committee meeting for July 29, 2026, to review Capricor Therapeutics’ Biologics License Application (BLA) for Deramiocel, an investigational cell therapy for the treatment of Duchenne muscular dystrophy. The application remains under review with a PDUFA target action date of August 22, 2026. Advisory Committee…
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FDA will hold Advisory Committee meeting on July 29th to review deramiocel for treatment of Duchenne
Capricor Therapeutics has shared that the US FDA will hold an Advisory Committee meeting on July 29, 2026, to discuss the company’s Biological License Application (BLA) seeking approval of deramiocel, an investigational cell therapy for the treatment of Duchenne. It will be possible to watch this meeting via live streaming, and more details will be…
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Update from Solid on microdystrophin gene therapy program
Solid Biosciences has provided an update to the community about SGT-003, their AAV-delivered microdystrophin gene therapy. In the Phase 1/2 INSPIRE DUCHENNE clinical trial, 53 boys have been dosed, ranging from 6 months to 10 years old. Solid has also started their Phase 3 IMPACT DUCHENNE trial, with sites currently enrolling in Australia and Canada. …
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Capricor Therapeutics Shares Positive Five-Year HOPE-2 OLE Data for Deramiocel in Duchenne
Today, Capricor Therapeutics announced positive five-year data from the company’s ongoing HOPE-2 open label extension (OLE) study of Deramiocel, Capricor’s novel cell therapy, in individuals living with Duchenne. According to Capricor, among the nine patients who remain enrolled in the HOPE-2 OLE study, treatment with Deramiocel continued to attenuate disease progression over five years, as…
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Solid Biosciences Shares Updates on SGT-003
Solid Biosciences has shared a new letter to the Duchenne community providing updates on the development of SGT-003, the company’s investigational gene therapy for Duchenne. Solid shared that the ongoing INSPIRE DUCHENNE Phase 1/2 clinical trial has dosed 53 patients ages 0 to 10 years old, including a 6-month-old child, who is the youngest known…
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Catching up on What’s Happening at PPMD’S 2026 Annual Conference
PPMD’s 2026 Annual Conference is in full swing! PPMD’s 2026 Annual Conference kicked off with opening remarks from Katherine Beaverson, MD, CEO of PPMD, who welcomed attendees, introduced the dedicated PPMD team, recognized the many individuals and partners who strengthen our community, and shared how PPMD is working every day to accelerate research, improve care,…
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MDA says DOJ memo threatens community care rights
The Department of Justice (DOJ) has released a memorandum arguing that states are not required to provide in-home or community-based care to people with disabilities, including those with muscular dystrophy (MD). The move drew sharp condemnation from the Muscular Dystrophy Association (MDA), which stated in a press release that it “will work tirelessly to protect…
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Avidity Biosciences Submits Biologics License Application to FDA for del-zota
Today, Avidity Biosciences announced that it has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) seeking approval of delpacibart zotadirsen (del-zota) for individuals living with Duchenne who are amenable to exon 44 skipping. The application is being reviewed through the FDA’s Accelerated Approval pathway and is supported by data…
