Lindsey Shapiro, PhD, science writer —

Dosing has been paused in a Phase 3 trial testing fordadistrogene movaparvovec in children with Duchenne muscular dystrophy (DMD) following the sudden death of a boy who had been previously treated with the gene therapy, its developer Pfizer announced in a letter to the community. Pfizer indicated…

Agamree (vamorolone) is now available for Duchenne muscular dystrophy (DMD) patients, ages 2 and older, in the U.S., where it will be dispensed through a specialty pharmacy network. The U.S. Food and Drug Administration (FDA) approved the next-generation steroid, expected to be more tolerable than standard…

The investigational cell therapy CAP-1002 has continued to slow declines in upper limb and heart function among boys and young men with Duchenne muscular dystrophy (DMD), according to two-year results from the HOPE-2 open label extension (OLE) study. The findings were presented at the recent 2024 Muscular Dystrophy…

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…