Janet Stewart, MSc,  —

Capricor Therapeutics presented positive six-month results from the HOPE trial of CAP-1002, a stem cell-based therapy for Duchenne muscular dystrophy (DMD), at the 22nd International Congress of the World Muscle Society, Oct. 3-7 in St. Malo, France. The poster is available on Capricor’s website. The Phase 1/2 HOPE trial…

Positive results were reported by Catabasis Pharmaceuticals from the open-label extension MoveDMD clinical trial evaluating edasalonexent (CAT-1004) in boys with Duchenne muscular dystrophy (DMD). A global Phase 3 trial of the investigative drug is projected for the first half of 2018. The new data were presented at…

In a recent webinar, Dr. Jerry Mendell with Nationwide Children’s Hospital rolled out plans for a Phase 1/2a clinical trial of a new gene therapy for Duchenne’s muscular dystrophy (DMD). Final go-ahead for the trial awaits U.S. Food and Drug Administration (FDA) approval of an Investigational New Drug (IND) application made for…

Many patients with Charcot-Marie-Tooth (CMT) disease, a subtype of muscular dystrophy, undergo surgeries to correct foot deformities. Yet a new study finds that surgeons vary significantly in their choice of procedures for the same operation. The study, “Prevalence and orthopedic management of foot and ankle deformities in Charcot Marie Tooth disease,”…

Experts provided an overview of the many approaches to counteract the loss of muscle mass, inflammation, fibrosis, calcium overload, oxidative stress, and ischemia (inadequate blood supply) in Duchenne muscular dystrophy (DMD), without actually targeting the cause, a mutation in the dystrophin gene. The review, “Pharmacological therapeutics targeting the secondary…

PTC Therapeutics announced that Emflaza (deflazacort), an approved treatment for all Duchenne muscular dystrophy (DMD) patients regardless of mutation, will be available in a matter of weeks in the U.S., and this time around with a starting list price of $35,000 a year — for a child weighing 25 kilograms, or about…

PTC Therapeutics, Inc. has entered into an asset purchase agreement with Marathon Pharmaceuticals to acquire all rights to Emflaza (deflazacort), which was approved in February by the U.S. Food Drug Administration to treat Duchenne muscular dystrophy patients 5 years and older, regardless of their genetic mutation. News about…