Margarida Azevedo, MSc,  —

Three grassroots foundations started by the families of boys with Duchenne muscular dystrophy (DMD) have come together to award a $100,000 research grant to the Kennedy Krieger Institute’s Center for Genetic Muscle Disorders to study potential therapies. The Kennedy Krieger Institute in Baltimore provides services for children…

Parent Project Muscular Dystrophy (PPMD), a nonprofit group dedicated to ending Duchenne muscular dystrophy (DMD), has named the Neuromuscular Research Center at the University of California Davis Department of Pysical Medicine and Rehabilitation a certified Duchenne care center. PPMD’s Certified Duchenne Care Center Program,…

Eighteen-year-old Brandon Martz will travel from Michigan to Washington, D.C., to raise awareness for muscular dystrophy (MD) and see the nation’s capital in one “final Dream trip” with the support of Dream Foundation. Brandon was diagnosed with Duchenne muscular dystrophy (DMD) when he was four and was…

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 rare degenerative disease. Edasalonexent is being evaluated in MoveDMD, an ongoing Phase 1/2 clinical trial (NCT02439216) consisting of…

Solid Biosciences of Cambridge, Mass., has completed the initial closing of a $50 million Series C round of financing to advance the company’s clinical portfolio of Duchenne muscular dystrophy (DMD) medications and programs. RA Capital Management and Bain Capital Life Sciences are the principal investors, with input also from RTW Investments, Foresite Capital,…

The nonprofit CureDuchenne organization has changed its website to better reflect its mission of improving the lives of people with Duchenne muscular dystrophy (DMD). CureDuchenne and the DMD community have taken heart from two recent Federal Drug Administration approvals. The initial one, in September 2016, was for the first-ever drug…

Hundreds seeking a cure for Duchenne muscular dystrophy will join April 8 at the University of Texas (UT) Golf Club for the Champions to CureDuchenne gala, now in its eighth year. The event, hosted by Quan Cosby, former wide receiver on the UT football team, and presented by…

Summit Therapeutics has confirmed plans for an extension stage of a Phase 2 clinical trial evaluating ezutromid as a treatment for boys with Duchenne muscular dystrophy (DMD). …

Data from RECENSUS, a medical chart review of boys with X-linked myotubular myopathy (XLMTM), demonstrates the significant medical burden for children living with the disease, a well as their families and caregivers. Alan Beggs, PhD, director of the Manton Center for Orphan Disease Research at Boston Children’s Hospital, presented data…

Akashi Therapeutics has received an all-clear signal from the U.S. Food and Drug Administration to resume developing a Duchenne muscular dystrophy (DMD) treatment. The FDA had ordered a hold on the development of delayed-release halofuginone (HT-100) after a clinical-trial participant began experiencing life-threatening adverse effects two weeks after beginning the therapy. HT-100 is…