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Scientists uncover why 1 steroid but not another slows growth in Duchenne boys
Scientists now have evidence to explain why treatment with the corticosteroid prednisone — but not the newer steroid Agamree (vamorolone) — drives bone loss in young boys with Duchenne muscular dystrophy (DMD) and may slow their growth. Using data from a clinical trial, the researchers found that prednisone, a standard treatment for DMD, significantly lowered…
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FDA Grants Priority Review to Potential Duchenne Treatment Del-Zota
As an early funder of Avidity Biosciences, we are pleased to share that Novartis has announced that the FDA has accepted the Biologics License Application (BLA) for delpacibart zotadirsen (del-zota) for Priority Review under the Accelerated Approval pathway. Del-zota, which was developed by Avidity, is being evaluated as a potential treatment for people with Duchenne who have a genetic…
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Novartis Shares Del-Zota Regulatory Update: FDA Accepts BLA for Priority Review
Novartis announced today that the U.S. Food and Drug Administration (FDA) has notified the company that the Biologics License Application (BLA) of del-zota (delpacibart zotadirsen) is sufficiently complete to permit a substantive review. According to Novartis’s September 8, 2026, community letter, the application has been granted Priority review and will be assessed under the Accelerated…
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What LGMD and a lengthy hospitalization reminded me about life
I have lived with limb-girdle muscular dystrophy (LGMD) type 2E/R4 for more than 40 years. Initially, apart from stretching and visiting a neuromuscular clinic twice a year, I didn’t think much about managing my symptom progression. So much has changed. In January 2024, I was forced to put my entire life on hold and be…
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Getting to Know You Survey
The post Getting to Know You Survey appeared first on Muscular Dystrophy News.
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Your Voice, Your Vote
Voting is more than a civic responsibility. It is a declaration of presence, power, and participation. The decisions made by elected officials touch nearly every aspect of our lives—from access to healthcare and medical research to community-based services, caregiver support, transportation, education, and employment. For people living with disabilities, however, participating in an election may…
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3 things that make my life as a Duchenne MD caregiver easier
With seven children — Lexi, 25; Max, 20; Chance, 19; Rowen, 17; Charlie, 15; Mary, 11; and Callie, 4; three of whom have Duchenne muscular dystrophy (DMD) — I have entered the zone of a veteran parent and caregiver. Max, Rowen, and Charlie were diagnosed with DMD in 2010 when they were just toddlers and…
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Treatment for FSHD named orphan drug, put on FDA fast track
The U.S. Food and Drug Administration (FDA) has granted both fast track and orphan drug designations to Scholar Rock‘s apitegromab as a potential treatment for facioscapulohumeral muscular dystrophy (FSHD), a disease type that characteristically affects muscles of the face and upper body. The experimental muscle-targeted therapy, which aims to improve motor function in FSHD, is…
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Guest Voice: 10 years after my son’s desperate plea to the FDA
On April 25, 2016, my son Billy stood at a podium in a hotel ballroom outside Washington, D.C., and told a room full of advisers from the U.S. Food and Drug Administration (FDA) something no 15-year-old should have to say out loud. “FDA, please don’t let me die early,” he said. This September marks 10…
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Community Bingo Challenge
The post Community Bingo Challenge appeared first on Muscular Dystrophy News.
