DMD

July 20, 2017

Sarepta Launches Exondys 51 Managed Access Program in Europe, North and South America

News

Sarepta Therapeutics has partnered with Idis Managed Access, a division of Clinigen Group, to develop a managed access program (MAP) for its lead therapy Exondys 51 (eteplirsen) for the treatment of Duchenne ... Read more

July 14, 2017

FDA Grants Orphan Designation to Mallinckrodt’s MNK-1411 for DMD

News

The U.S. Food and Drug Administation has granted orphan designation to Mallinckrodt Pharmaceuticals‘ drug candidate MNK-1411 for treatment of Duchenne Muscular Dystrophy (DMD). MNK-1411 is an injection composed of a formulation ... Read more

July 7, 2017July 7, 2017

Experts Review Current and Potential Treatments for Duchenne Muscular Dystrophy

News

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 ... Read more

June 26, 2017June 26, 2017

Edasalonexent Could Reduce Functional Decline in Boys with DMD, MoveDMD Phase 2 Results Suggest

News

Results of the Phase 2 MoveDMD trial showed that Catabasis Pharmaceuticals’ candidate drug edasalonexent (CAT-1004) can reduce the rate of functional decline in boys ages 4–7 with Duchenne muscular dystrophy (DMD). The recent ... Read more

June 12, 2017June 12, 2017

Sarepta Recruits Duchenne Boys for Phase 3 ESSENCE Trial to Test 2 Potential DMD Therapies

Duchenne Muscular Dystrophy News, News

Sarepta Therapeutics is recruiting up to 99 boys with Duchenne muscular dystrophy (DMD) to test two new compounds, SRP-4045 and SRP-4053, in a Phase 3 clinical trial. The ESSENCE study is ... Read more

May 19, 2017

CAP-1002 Positive Results Support New Clinical Trial

News

Positive results from the HOPE-Duchenne clinical trial support further clinical development of Capricor Therapeutics’ therapy candidate CAP-1002 for treatment of cardiac anomalies caused by Duchenne Muscular Dystrophy (DMD). The company ... Read more

April 28, 2017April 28, 2017

Stem Cell Therapy CAP-1002 Shows Promise in Treating DMD-linked Heart Disease

News

CAP-1002, developed by Capricor Therapeutics, safely and effectively treats cardiac anomalies associated with Duchenne muscular dystrophy (DMD). So says a clinical study whose preliminary results formed the basis of an April ... Read more

April 11, 2017April 11, 2017

Duchenne Muscular Dystrophy Bill SB 643 Passes California Senate Health Committee By 8-0 Vote

News

CureDuchenne has announced its enthusiastic support and sponsorship of California Senate bill SB 643, which adds Duchenne muscular dystrophy to California’s Genetically Handicapped Persons Program (GHPP) list of eligible medical conditions. The ... Read more

April 6, 2017April 6, 2017

Catabasis Reports Some Setbacks in Clinical Trial of Edasalonexent (CAT-1004) for Duchenne

News

Catabasis Pharmaceuticals is facing challenges in its efforts to develop Edasalonexent (CAT-1004) — an oral drug that targets NF-kB, a protein activated in Duchenne muscular dystrophy (DMD) — to treat the ... Read more

March 22, 2017March 22, 2017

PPMD’s ‘Run For Our Sons’ Fundraising Events Can Use Your Help

News

Parent Project Muscular Dystrophy (PPMD) is promoting a series of running events this year to raise money for research to help end Duchenne muscular dystrophy (DMD). The next Run ... Read more