News

When his daughter, Meredith Huml, was diagnosed — finally — with facioscapulohumeral (FSHD) muscular dystrophy in 2004, Raymond Huml immediately jumped into action, researching everything he could about the disease and reaching out to the FSHD Society. “We went to the FSHD Society and looked up what they…

A young man with Duchenne muscular dystrophy (DMD), who was participating in Pfizer’s Phase 1B open-label clinical trial evaluating its gene therapy candidate PF-06939926, has died. The patient has only been identified as a young man who was not able to walk. In a statement, Pfizer expressed…

A branch of the U.S. Food and Drug Administration (FDA) known as the Division of Cardiology and Nephrology has given positive feedback to Stealth BioTherapeutics‘ program for Duchenne muscular dystrophy (DMD). In a pre-investigational new drug (pre-IND) meeting, the division — called the DCN — agreed to the design of…

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…

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 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…

The show’s name may be “Ordinary Joe,” but this NBC drama series is proving extraordinary for its authentic depiction of a character with a disability, portrayed by John Gluck, 13, who has collagen VI muscular dystrophy. The show follows Joe Kimbreau, played by actor James Wolk, in three different…

Treatment with vamorolone continued to show a good safety profile and sustained efficacy across functional assessments after almost one year in boys with Duchenne muscular dystrophy (DMD), according to 48-week results of the Phase 2b VISION-DMD clinical trial. Data also showed no loss of efficacy in boys who switched…

Author and advocate Elijah J. Stacy is using what he has learned through adversity in battling Duchenne muscular dystrophy (DMD) to help others overcome their own challenges. DMD is a progressive muscle wasting disease that slowly robs a person’s muscle strength. In Stacy’s case, he lost the ability to…

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…