News

May 21, 2018May 21, 2018

FDA Grants Rare Pediatric Disease Status to Myonexus’ MYO-101 for LGMD 2E

News

The U.S. Food and Drug Administration recently granted rare pediatric disease designation to Myonexus Therapeutics’ experimental gene therapy MYO-101 for the treatment of limb girdle muscular dystrophy (LGMD) type 2E, or ... Read more

May 18, 2018May 18, 2018

Myoda (Sarconeos) Earns FDA’s Orphan Drug Designation for Duchenne MD

News

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to investigational therapy Sarconeos for Duchenne muscular dystrophy (DMD), Biophytis, the therapy’s developer, announced. The company also has filed ... Read more

May 17, 2018May 17, 2018

‘Rare Impact Awards’ Dinner Marks Orphan Drug Act and NORD at 35

News

The National Organization for Rare Disorders (NORD) will celebrate the 35th anniversary of both the 1983 Orphan Drug Act and NORD’s founding at a dinner tonight in Washington, D.C. ... Read more

May 16, 2018

#ECRD2018 – EU Must Do More for Rare Disease Patients, Eurordis Leaders Say

News

The European Union isn’t doing enough to protect the 30 million or so people with rare diseases who live in its 28 member countries, officials meeting last week in ... Read more

May 16, 2018May 16, 2018

Sarepta, Myonexus Collaborating to Advance Gene Therapies for LGMD

News

Sarepta Therapeutics has established an exclusive collaboration agreement with Myonexus Therapeutics to develop five new therapies targeting various forms of limb-girdle muscular dystrophy (LGMD). These include three clinical and two ... Read more

May 14, 2018May 14, 2018

New Exon 51 Skipping Therapy in Phase 1 Safety Trial Explained by Sarepta CEO in Webinar

News

Exondys 51 (eteplirsen), an RNA-targeted therapy marketed by Sarepta Therapeutics, is the first approved treatment targeting the underlying cause of Duchenne muscular dystrophy (DMD). Sarepta is continuing to refine its RNA-targeted ... Read more

May 11, 2018May 11, 2018

Approved Anti-inflammatory Seen to Increase Muscle Strength and Function in DMD Mice in Study

News

Celebrex (celecoxib), an FDA-approved anti-inflammatory and pain reliever marketed by Pfizer, showed an ability to improve strength and function in leg, heart and diaphragm muscles of an animal model of Duchenne ... Read more

May 8, 2018May 8, 2018

PPMD Marks 10th Year of Duchenne Registry, Largest Repository of DMD Patient Data

News

The Duchenne Registry, formerly known as DuchenneConnect, marks its 10th anniversary as the world’s largest repository of patient-reported data on Duchenne muscular dystrophy (DMD): 4,200 people from more than 100 ... Read more

May 7, 2018May 7, 2018

Capricor Recruiting Duchenne Patients for Phase 2 Trial of CAP-1002

News

Capricor Therapeutics’ Phase 2 clinical trial to evaluate the safety and effectiveness of CAP-1002 in boys and young men with Duchenne muscular dystrophy (DMD) has started recruiting participants. The HOPE-2 ... Read more

May 4, 2018May 4, 2018

Sarepta Therapeutics Launches ‘Route 79, the Duchenne Scholarship Program’

News

Duchene muscular dystrophy (DMD) patients now have a new scholarship program that could help them pursue their post-high school education goals. Sarepta Therapeutics recently launched “Route 79, the Duchenne ... Read more

Pin It on Pinterest