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The National Organization for Rare Disorders (NORD) is seeking participants for its survey-based study Living Rare, which aims to better understand the real-world lived experiences of people in the U.S. with rare diseases. Living Rare, the first large-scale study of its kind in the U.S., seeks to capture the…

Asklepios Biopharmaceutical (Askbio) has dosed the first participant in the second group of a Phase 1/2 trial testing AB-1003, an experimental gene therapy for limb-girdle muscular dystrophy (LGMD) type 2I/R9. The Phase 1/2 LION-CS101 trial (NCT05230459) is assessing AB-1003’s safety and tolerability in adults with LGMD2I/R9. Enrollment…

The U.S. Food and Drug Administration (FDA) has accepted Capricor Therapeutics’ application seeking approval of deramiocel — a  cell therapy developed by the biotech company to treat heart muscle disease in people with Duchenne muscular dystrophy (DMD) — and granted it priority review that will speed the agency’s…

Note: This story has been updated March 6, 2025, to correct a quote from Mindy Henderson, MDA vice president of disability outreach and empowerment. The Muscular Dystrophy Association (MDA) is once again hosting its annual gathering, the MDA Clinical & Scientific Conference. The 2025 event will take place…

Preventive treatment with standard heart medications — when given before the onset of cardiac troubles — extends survival among males with Duchenne muscular dystrophy (DMD), according to data from the U.S.-based Muscular Dystrophy Surveillance, Tracking and Research Network, known as MD STARnet. However, despite finding such benefits with prophylactic,…

The U.S. Food and Drug Administration (FDA) has given Entrada Therapeutics permission to launch a Phase 1b clinical trial to test ENTR-601-44, the company’s experimental exon-skipping therapy, in adults with Duchenne muscular dystrophy (DMD) who carry a mutation that’s amenable to exon 44 skipping. Entrada had sought…

Adolescents and young adults with Duchenne (DMD) or Becker muscular dystrophy (BMD) who have more siblings typically report being better prepared to transition to adult life, a new study has found. Findings show people with DMD or BMD had the most difficulty with activities of daily living, education,…

Five people with oculopharyngeal muscular dystrophy (OPMD) — a type of muscular dystrophy marked by muscle weakness affecting the face and throat — have thus far been treated with the gene therapy candidate BB-301 in an ongoing clinical trial, treatment developer Benitec Biopharma said in a…

A Phase 1 clinical trial testing SAT-3247, an oral treatment candidate for Duchenne muscular dystrophy (DMD), is now done enrolling healthy volunteers, its developer Satellos Bioscience announced. “The completion of healthy volunteer enrollment in our Phase 1 trial marks a significant milestone in our mission to develop transformative…

A fundraiser through the online role-playing game World of Warcraft has raised more than $2 million to support CureDuchenne, an organization dedicated to combating Duchenne muscular dystrophy (DMD). The fundraiser, which launched last year in partnership with Warcraft’s maker Blizzard Entertainment, is the largest contribution from a…