News

CITGO raised $2.3 million this year to help advance research, improve patient care, and bolster advocacy efforts for the neuromuscular community, continuing its long-standing support for the Muscular Dystrophy Association (MDA), . Throughout the year, employees of the Houston, Texas-based petroleum company, along with vendors, contractors, marketers,…

The U.S. Food and Drug Administration (FDA) has given priority review Sarepta Therapeutics‘ application seeking accelerated approval of SRP-9001 (delandistrogene moxeparvovec), the company’s experimental gene therapy for Duchenne muscular dystrophy (DMD) in patients who can walk. An FDA decision is expected on or before May 29, 2023. “We are delighted…

Blocking a calcium channel called TRPC6 prolonged survival in a mouse model of Duchenne muscular dystrophy (DMD) by improving skeletal and heart muscle function and reducing bone deformities, a study showed. Its results support clinical trials of TRPC6 inhibitors in Duchenne patients as “a novel DMD therapy,” the researchers wrote,…

Analyzing the type of mutation in the DMD gene combined with assessing motor milestones may help better distinguish Duchenne muscular dystrophy (DMD) from Becker muscular dystrophy (BMD) early in life, a study found. Combining both approaches had better predictive power than using either alone, and could mean that children will…

PGN-EDO53, an experimental therapy designed to treat Duchenne muscular dystrophy (DMD) in patients amenable to exon 53 skipping, showed promising effects in preclinical studies, the therapy’s developer PepGen announced. The company also announced promising results from cell experiments for two other investigational exon-skipping therapies, PGN-EDO45 and PGN-EDO44.

Sarepta Therapeutics plans to begin a clinical trial to test the antibody-cleaving therapy imlifidase as a pre-treatment for SRP-9001 (delandistrogene moxeparvovec) — its experimental gene therapy for Duchenne muscular dystrophy (DMD) — in patients with pre-existing antibodies against the gene therapy’s viral carrier. Preclinical work conducted by…

The U.S. Food and Drug Administration (FDA) has granted fast track designation to Dyne Therapeutics’ DYNE-251 for the treatment of people with Duchenne muscular dystrophy (DMD) who have mutations amenable to exon 51 skipping. Fast track status is intended to accelerate a therapy’s development and expedite…

Santhera and ReveraGen have completed their rolling application asking the U.S. Food and Drug Administration (FDA) to approve the dissociative corticosteroid vamorolone to treat Duchenne muscular dystrophy (DMD). Rolling applications allow companies to submit individual sections of the application as soon as they are completed, rather than waiting…

Parent Project Muscular Dystrophy (PPMD) and Duchenne UK will jointly award up to $1 million to two research projects that seek to develop new treatments for Duchenne muscular dystrophy (DMD), especially those involving cell and gene therapy. The call for proposals for projects, which are expected…

Treatment with Viltepso (viltolarsen) helps preserve motor function in boys with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping, according to four-year data from an open-label extension study. “These data represent the longest clinical experience of an exon 53 skipping therapy for the treatment of Duchenne…