News

Elamipretide as Add-on May Boost Efficacy of Exon-skipping Therapies

Elamipretide, an investigational therapy designed to increase the activity of mitochondria, may boost the effectiveness of exon-skipping therapies for Duchenne muscular dystrophy (DMD), its developer, Stealth BioTherapeutics, reported. “Our new preclinical data increases our optimism that elamipretide may help address the unmet need for therapies in DMD,” Reenie…

UK Panel Expands Care Guidelines for Adults With DMD

The U.K. Adult North Star Network (ANSN) has developed a set of guidelines for best care management of adults with Duchenne muscular dystrophy (DMD) based on consensus among experts in the field. The guidelines were outlined in a report, “Adult North Star Network (ANSN): Consensus Guideline…

Atamyo Cleared in UK to Test ATA-100 for Limb-Girdle MD Type R9

Atamyo Therapeutics has been given the go-ahead to start clinical testing of ATA-100, the company’s investigational gene therapy for a specific form of limb-girdle muscular dystrophy (LGMD) called type R9 (LGMDR9). With approval from the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA), Atamyo now plans to start dosing…

DMD Gene Therapy Candidate RGX-202 Earns Orphan Drug Status

RGX-202, a one-time experimental gene therapy for the treatment of Duchenne muscular dystrophy (DMD), has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA). The designation is given to investigational therapies to treat rare medical conditions or diseases that affect fewer than 200,000 people…

Santhera, ReveraGen Advancing Plans for Vamorolone Application

Santhera Pharmaceuticals and ReveraGen BioPharma are gearing up to submit a new drug application (NDA) asking for U.S. approval of vamorolone as a treatment for Duchenne muscular dystrophy (DMD). The companies announced Tuesday they had completed a pre-NDA meeting with the U.S. Food and Drug Administration (FDA).