Steve Bryson, PhD,  science writer—

The Critical Path Institute (C-Path) is launching a task force to advance the development of therapies for limb-girdle muscular dystrophy (LGMD) and will lead the efforts of its members to find a new treatment to, among other goals, slow or stop the progression of the muscle-wasting disease. Formed…

A noninvasive quantitative MRI, or qMRI, was found to detect early muscle abnormalities among people with limb-girdle muscular dystrophy type R1 (LGMDR1), according to a small study from Europe. Many of the qMRI findings correlated with clinical assessments of muscle function and patient-reported activities. “Our findings revealed alterations in…

Avidity Biosciences‘ delpacibart zotadirsen, or del-zota for short — formerly called AOC 1044 — was found to safely increase levels of dystrophin production to 25% of normal in people with Duchenne muscular dystrophy (DMD), according to new data from the EXPLORE44 trial. In the Phase 1/2 clinical…

One year of losmapimod taken twice a day improved or stabilized upper limb function and muscle strength in adults with facioscapulohumeral muscular dystrophy (FSHD), according to an open-label pilot study. Blood and muscle tests also showed the therapy candidate successfully engaged with its intended target and had a…

After three years of treatment with Capricor Therapeutics’ experimental cell therapy CAP-1002, people with Duchenne muscular dystrophy (DMD) continue to show benefits in arm and heart function, new data from the HOPE-2 open-label extension (OLE) study shows. Topline data from the Phase 3 HOPE-3 trial (NCT05126758), which…

A $250,000 award from the Parent Project Muscular Dystrophy (PPMD) will support the establishment of Baby Duchenne, a collaborative clinical research network for babies with Duchenne muscular dystrophy (DMD) diagnosed via newborn screening (NBS) programs in New York state. Under the direction of Bo Hoon Lee, MD, from…

Researchers have discovered a new, unrecognized type of muscular dystrophy that’s caused by inherited mutations in the SNUPN gene, a study reports. Most people who carry the mutations develop symptoms of muscle weakness before age 2, and the muscles of the upper arms and legs are mainly affected. “This…

Up to eight years of Exondys 51 treatment (eteplirsen or AVI-4658) extended survival in Duchenne muscular dystrophy (DMD) patients over a wide range of ages, a long-term study concluded. Patients treated for longer periods had the lowest risk of death. The study, “Survival among patients…

Regulators in France and Italy have given Atamyo Therapeutics the green light to launch a clinical trial testing ATA-200, an investigational gene therapy for children with a type of limb-girdle muscular dystrophy called LGMD2C or LGMDR5. The multicenter, dose-escalation Phase 1b study (NCT05973630) — which will assess…

A cell-based study revealed the biological mechanism behind the increased tendency for people with myotonic dystrophy type 2 (DM2) to develop autoimmune diseases. Researchers found that the genetic defect that causes DM2, called a repeat expansion, indirectly triggered an abnormal, antiviral immune response in patient cells. “That was our…