Lindsey Shapiro, PhD,  science writer—

DYNE-251 and DYNE-101, Dyne Therapeutics’ investigational therapies for forms of muscular dystrophy, appear safe and are showing signs of preliminary efficacy in Phase 1/2 clinical trials. These proof-of-concept data, announced by Dyne in January, were discussed in two oral presentations at the recent 2024 Muscular Dystrophy…

Among ambulatory boys with Duchenne muscular dystrophy (DMD), a one-time treatment with investigational gene therapy fordadistrogene movaparvovec has helped preserve functions and increase muscle volume for three years, especially in the youngest patients. That’s according to updated analyses from a Phase 1b trial (NCT03362502), in which the…

HuidaGene Therapeutics’ investigational gene-editing therapy, called HG302, for Duchenne muscular dystrophy (DMD) has been granted a rare pediatric drug designation by the U.S. Food and Drug Administration (FDA). This status is intended to incentivize companies to develop treatments for rare and serious or life-threatening diseases affecting people under…

In October, the U.S. Food and Drug Administration (FDA) approved Agamree (vamorolone) for treating Duchenne muscular dystrophy (DMD) patients ages 2 and older, offering them a more tolerable, but similarly efficacious, oral corticosteroid option. Clinical trial evidence indicates Agamree might ease muscle damage and minimize the side effects…

The U.S. Food and Drug Administration (FDA) has approved vamorolone for treating people with Duchenne muscular dystrophy (DMD), ages 2 and older. The therapy will be marketed as Agamree. “We strongly believe that this novel steroid has the transformational potential to make a significant difference for patients living with…

Researchers have created a three-dimensional (3D) artificial muscle model, derived from patient cells, that accurately replicates the damage observed in people with Duchenne muscular dystrophy (DMD). This new model, the scientists believe, will enable more efficient testing of treatments that could potentially reverse such muscle damage. The findings were…

Ahead of pending regulatory decisions for vamorolone — a treatment for Duchenne muscular dystrophy (DMD) now under review in the U.S. and Europe — Catalyst Pharmaceuticals has fully acquired the commercial rights to the therapy in North America. The finalized deal comes about a month after Catalyst…

The Muscular Dystrophy Association (MDA) has launched the Gene Therapy Support Network (GTx) that will offer resources and guidance about approved gene therapies for people living with neuromuscular diseases. Its launch comes shortly ahead of a possible U.S. approval of SRP-9001 (delandistrogene moxeparvovec), Sarepta Therapeutics‘ experimental…

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…

Pelvic floor disorders, including urinary and anal incontinence, are common in women with myotonic dystrophy type 1 (DM1) and a cause of significant distress, according to a recent study in Canada. “This study highlights the importance of screening these symptoms in a clinical setting and will help develop interventions…