Margarida Azevedo, MSc,  —

Los Angeles-based Capricor Therapeutics has been awarded nearly $3.4 million to support its Phase 1/2 HOPE-Duchenne clinical trial. The grant is from CIRM — the California Institute for Regenerative Medicine — as part of the CIRM 2.0 program, a joint initiative meant to advance the development of treatments based on stem cells for people…

On April 9, hundreds of Texans will meet at the University of Texas Golf Club in Austin for the seventh annual Champions to CureDuchenne gala to raise both awareness for Duchenne muscular dystrophy (DMD) and funds to advance research to further the mission of CareDuchenne, which is to find a cure…

Summit Therapeutics plc announced the publication of a study into new imaging techniques that appear to reliably and reproducibly measure utrophin protein levels and muscle fiber regeneration in muscle biopsies in Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) patients. The study is important to the company because one of its…

PTC Therapeutics has announced it will submit the results its Phase 3 ACT DMD clinical trial for review by Health Canada as part of the New Drug Submission (NDS) for Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). DMD is a progressive disease that mostly affects male patients…

Congenica, a global leader in the development of genome-based diagnostic technologies, has taken on a new project: to help a woman named Jill Viles with a serious muscle-wasting disease linked to a muscle-bound phenotype by offering to sequence her and her family’s genome. Viles and her family are affected by…

Northbrook, Illinois-based Marathon Pharmaceuticals, a biopharma that focuses on the development of medicines to treat rare diseases with no current treatment options, has announced it is expanding patient access to its investigational medication deflazacort through its ACCESS DMD program. The program will allow participating physicians to obtain and prescribe deflazacort, which is…

Ways of better addressing the high cost of breakthrough treatments that either are or might soon be available for a range of chronic and rare diseases, from certain cancers to Duchenne muscular dystrophy (DMD) or Becker’s muscular dystrophy (BMD), was the subject of recent research — with the authors suggesting that healthcare…

Tarix Orphan LLC recently announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to TXA127, the company’s lead candidate for the treatment of laminin-deficient congenital muscular dystrophy (LAMA2 MD). Early-onset LAMA2-related muscular dystrophy (MD) occurs in nearly one in every 30,000 people and is responsible for…

Catabasis Pharmaceuticals and the Muscular Dystrophy Association (MDA) have announced a partnership that will support Part B of the MoveDMD clinical trial, currently evaluating the effectiveness and safety of Duchenne muscular dystrophy (DMD) treatment candidate CAT-1004 in children. CAT-1004, believed to be a disease modulator regardless of mutation, is…

Jiffy Lube International has raised more than $1 million dollars in a campaign to support the Muscular Dystrophy Association (MDA). The company holds more than 2,000 franchised oil change service centers and has partnered with MDA for the fourth year to support children and adults who suffer from muscular dystrophy,…