News

May 17, 2019May 17, 2019

Sarepta Acquires Rights to Novel Gene Therapy Candidate to Treat Limb-Girdle MD

News

Sarepta Therapeutics is acquiring an investigational gene therapy program focused on calpain-3 for the treatment of limb-girdle muscular dystrophy type 2A (LGMD2A). The candidate therapy was developed by the Research ... Read more

May 15, 2019

Rare Diseases Constitute a ‘Public Health Issue,’ NCATS Director Warns

News

With 250 rare diseases newly identified every year, scientists can barely keep up — even as the healthcare system fails millions of Americans whose rare diseases have already been ... Read more

May 15, 2019May 15, 2019

Duchenne Teen Hits the Runway at Las Vegas Fashion Show

News

Joey Chorzewski has Duchenne muscular dystrophy, a neuromuscular disease that makes him different from most of his peers in Marshfield, Massachusetts. But in other ways — like wanting to ... Read more

May 13, 2019May 13, 2019

#AANAM – FSHD Patients ‘Very Enthusiastic’ About Taking Part in Clinical Trials, Research Suggests

News

People with facioscapulohumeral muscular dystrophy (FSHD) are “very enthusiastic” about participating in clinical trials and studies, according to researchers, who report that foot and leg, and shoulder and arm ... Read more

May 13, 2019May 13, 2019

PPMD Launches Pilot Duchenne Newborn Screening Program in NY

News

Parent Project Muscular Dystrophy (PPMD) has launched a pilot program to screen newborns in New York state for Duchenne — one of the most common forms of muscular dystrophy. ... Read more

May 10, 2019May 10, 2019

#AANAM — New TTN Gene Variants Potentially Linked to Limb-girdle MD, Study Says

News

Limb-girdle muscular dystrophy (LGMD) may be caused by previously unidentified TTN gene variants that are also associated with heart muscle disease, according to a study. Findings of the study, ... Read more

May 8, 2019May 8, 2019

#AANAM — Very Young Boys with DMD May Have Measurable Early Signs of Motor Skill Delay, Study Suggests

News

Very young boys with Duchenne muscular dystrophy (DMD) have measurable differences in motor skills, which could have implications for the early detection of DMD and for evaluating how treatments ... Read more

May 8, 2019May 8, 2019

#AANAM — Phase 1b/2 Trial Of RG6206 in Boys With DMD Supports Safety, Positive Muscle Effects

News

RG6206 (BMS-986089), a blocker of muscle growth-inhibitor myostatin, is safe to use in boys with Duchenne muscular dystrophy (DMD), results from a Phase 1b/2 trial show. A Phase 2/3 ... Read more

May 6, 2019

Gene Therapy Candidate GALGT2 Found Safe in Parent Project-Funded Toxicology Study

News

The investigational gene therapy GALGT2 for Duchenne muscular dystrophy (DMD) was found to be safe in a preclinical study conducted at the Research Institute at the Nationwide Children’s Hospital, ... Read more

May 3, 2019

EGF Protein Preserves Muscle Strength, Promotes Muscle Regeneration in DMD Mice, Study Shows

News

A protein called epidermal growth factor (EGF) can help preserve muscle strength and increase muscle regeneration in a mouse model of Duchenne muscular dystrophy (DMD), a finding that may ... Read more

Pin It on Pinterest