News

September 17, 2020September 17, 2020

Real-world Data Could Replace Placebo Groups in DMD Clinical Trials, Study Indicates

News

Real-world and natural history data could be used to supplement, or perhaps replace, the use of a placebo in future clinical trials in people with Duchenne muscular dystrophy (DMD), ... Read more

September 15, 2020September 15, 2020

Sarepta Poised to Start Next Trial of SRP-9001 for Duchenne MD

News

Sarepta Therapeutics has completed a meeting with the Office of Tissues and Advanced Therapies (OTAT) that clarified regulatory requirements to start the company’s next clinical trial of SRP-9001, an ... Read more

September 10, 2020September 10, 2020

Differences in Key Proteins in DMD Muscle Cells Favor Fat Formation, Study Says

News

An imbalance in cells producing proteins called aldehyde dehydrogenases (ALDHs) — favoring fat formation in tissue — is evident in the muscles of people with Duchenne muscular dystrophy (DMD), ... Read more

September 8, 2020September 8, 2020

Santhera Acquires Global Rights to DMD Therapy Vamorolone

News

Santhera Pharmaceuticals has obtained the worldwide rights to vamorolone (VBP15), an experimental therapy for Duchenne muscular dystrophy (DMD), the company announced. The company has reached an agreement with ReveraGen Biopharma, ... Read more

September 3, 2020September 3, 2020

September is Duchenne Action Month

News

September is Duchenne Action Month, an awareness, fund-raising, and educational campaign supporting people with Duchenne muscular dystrophy (DMD) Hosted by Parent Project Muscular Dystrophy (PPMD), each week of the ... Read more

September 2, 2020September 2, 2020

Same But Different Photo Contest Celebrates People With Rare Diseases

News, syndicated

Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme ... Read more

September 1, 2020September 1, 2020

Virtual Events Mark MDA’s Muscular Dystrophy Awareness Month

News

This year, the Muscular Dystrophy Association (MDA) will celebrate MDA National Muscular Dystrophy Awareness Month, held every September, with a series of online events. The events will be held virtually ... Read more

August 27, 2020August 27, 2020

Casimersen, Exon 45 Skipping Duchenne Therapy, Under FDA Priority Review

News

An application requesting the approval of casimersen (SRP-4045) to treat Duchenne muscular dystrophy (DMD) amenable to exon 45 skipping has been accepted and placed under priority review by the U.S. Food and Drug ... Read more

August 25, 2020August 25, 2020

Viltepso Commercially Available in the US for DMD Treatment

News

NS Pharma‘s Viltepso (viltolarsen) is now commercially available in the U.S. for people with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping. The FDA conditionally approved Viltepso earlier this month, ... Read more

August 24, 2020August 24, 2020

Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input

News, syndicated

Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient ... Read more

Pin It on Pinterest