GIVI-MPC, a stem cell therapy to create new muscle in Duchenne muscular dystrophy (DMD), has been designated an orphan drug by the U.S. Food and Drug Administration (FDA). Orphan drug status encourages the development of therapies for rare diseases — those affecting fewer than 200,000 people in the U.S.
Thousands of retailers across the U.S. are helping the Muscular Dystrophy Association (MDA) kick off MDA Shamrocks, its annual fundraising campaign in support of those living with muscular dystrophy, amyotrophic lateral sclerosis, and other neuromuscular disorders (NMDs). Money raised through the effort — said to be…
AOC 1020, Avidity Biosciences’ investigational therapy for facioscapulohumeral muscular dystrophy (FSHD), has been granted orphan drug status by the U.S. Food and Drug Administration (FDA). The designation is intended to encourage the development of therapies for rare diseases, those affecting fewer than 200,000 people in the U.S. It provides…
Nearly half of the 24 children with myotonic dystrophy type 1 (DM1) in a French study showed signs of poorer nighttime respiration, reflected by rising carbon dioxide (CO2) levels. These increases in CO2 did not significantly associate with the presence of sleep apnea — when breathing repeatedly starts and…
Five of seven clinical milestones in managing Duchenne muscular dystrophy (DMD) occurred significantly later for Black males than for white males, a new study reports. These gaps appeared to stem from the initial delays in the first evaluation, which occurred for Black children 2.3 years later than for white…
New research into a mechanism underlying Duchenne muscular dystrophy (DMD) — which revealed a chemical modification in gene activity — has shed light on potential treatments for the muscle-wasting disease, and may aid in diagnosis, according to scientists. DMD, the study showed, is characterized by a specific pattern of…
Whole-body muscle MRI correlates with functional measures in children with facioscapulohumeral muscular dystrophy (FSHD) and might help evaluate disease severity, according to a study. “This study demonstrates that MR muscle imaging is a potential biomarker of disease staging in pediatric FSHD,” the researchers wrote in “Correlation between whole body…
A new dominant-negative tumor necrosis factor (DN-TNF) treatment reduced muscle damage and promoted muscle regeneration in a mouse model of Duchenne muscular dystrophy (DMD), according to INmune Bio. The research is part of a collaboration between INmune and Armando Villalta, PhD, a professor at the University California, Irvine…
After 18 months, treatment with the experimental cell therapy CAP-1002 continues to show beneficial effects on upper limb function in people with Duchenne muscular dystrophy (DMD), according to new data from the open-label extension phase of the HOPE-2 study. The new trial data also suggests a consistent safety…
Regenxbio has started recruiting patients for the Phase 1/2 clinical trial to assess the safety and effectiveness of RGX-202, an investigational gene therapy for Duchenne muscular dystrophy (DMD). The trial, called AFFINITY DUCHENNE (NCT05693142), is expected to enroll 18 DMD patients ages 4 to 11 who…
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