Lindsey Shapiro, PhD,  science writer—

Delpacibart zotadirsen (del-zota), an investigational exon-skipping therapy from Avidity Biosciences, is demonstrating the potential to reverse disease progression in boys and young men with Duchenne muscular dystrophy (DMD) amenable to exon 44 skipping, or DMD44. That’s according to one-year data from the Phase 1/2 EXPLORE44 trial (NCT05670730)…

Researchers have developed a detailed molecular model for the structure of type VI collagen and identified where genetic mutations linked to certain forms of muscular dystrophy (MD) might influence the protein. Findings from their study could ultimately help scientists better understand the mechanisms underlying types of MD that affect…

Ifetroban, Cumberland Pharmaceuticals’ investigational oral therapy, improved heart function and reduced cardiac damage biomarkers in people with Duchenne muscular dystrophy (DMD), according to new trial data. These findings from the Phase 2 FIGHT DMD trial (NCT03340675) were presented at the Parent Project Muscular Dystrophy (PPMD) annual…

The one-time gene therapy Elevidys (delandistrogene moxeparvovec-rokl) was associated with stabilizations in motor function for boys with Duchenne muscular dystrophy (DMD) who were treated when they were 8 to 9 years old, according to new trial analyses. Boys with DMD at this age are typically expected to show…

Capricor Therapeutics’ investigational therapy deramiocel, formerly CAP-1002, which is being considered for approval in the U.S., continued to show an ability to preserve upper limb function in boys and men with Duchenne muscular dystrophy (DMD) over the long term. That’s according to analyses of about five years…

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…

The investigational therapy DYNE-101 worked as expected and led to functional improvements for people with myotonic dystrophy type 1 (DM1) in a clinical trial, according to an update from developer Dyne Therapeutics. The Phase 1/2 ACHIEVE trial (NCT05481879) will now enroll a new group of participants…

A gene-editing therapy designed to correct a defect in the DYSF gene — one that’s associated with a form of limb-girdle muscular dystrophy, or LGMD — was found to restore dysferlin protein levels and help regenerate muscle tissue in a mouse model of the disease. The researchers are now seeking…

Arrakis Therapeutics’ investigational RNA-targeted small molecule (rSM) therapies were found to work as intended in preclinical studies and eased symptoms in a mouse model of myotonic dystrophy type 1 (DM1). The company presented the findings at the Cell Symposia: Chemical Biology in Drugging the Undrugged conference, held earlier…

A single low dose of the investigational gene therapy BB-301 helped with swallowing for the first two people with oculopharyngeal muscular dystrophy (OPMD) treated in a Phase 1b/2a clinical trial, according to an update from the therapy’s developer, Benitec Biopharma. Trial findings to date, which include reasonable…