Uncategorized

Edgewise Therapeutics, Inc., has announced positive topline results from the double-blind, randomized, placebo-controlled Phase 2 CANYON trial of sevasemten in individuals with Becker muscular dystrophy. Sevasemten is an orally administered small molecule inhibitor designed to protect muscle against contraction-induced damage in muscular dystrophies. According to Edgewise, the trial met its primary endpoint of change from…

Know More

As an early funder of Edgewise, CureDuchenne is pleased to share that the Phase 2 Trial of sevasemten in Becker muscular dystrophy met its primary endpoint of reduction in creatine kinase (CK), a biomarker associated with skeletal muscle damage.  Individuals treated with sevasemten also showed stabilization of NSAA, with a trend towards improvement at 12…

Know More

The investigational oral therapy sevasemten significantly reduced markers of muscle damage in people with Becker muscular dystrophy (BMD) treated in a Phase 2 trial, according to top-line results announced by the therapy’s developer, Edgewise Therapeutics. “This landmark study presents compelling biomarker data and promising signals that suggest the potential for functional stabilization with administration of…

Know More

A novel drug holds promise for treating Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes severe muscle degeneration. Researchers have discovered that an experimental compound called K884 can boost the natural repair abilities of muscle stem cells. Current treatments can slow muscle damage, but don’t address the root problem.

Know More

I’m more than a caregiver, but it took a recent life change for me to realize that. I’m a mom to seven children I share with my husband, Jason, including three of our sons — Max, 19, Rowen, 15, and Charlie, 13 — who live with Duchenne muscular dystrophy (DMD). Their DMD diagnoses happened in…

Know More

In 1984, the word “Duchenne” entered my world. The progress we have made since then is astounding. For decades, we have united in our unwavering commitment to end Duchenne, and with recent achievements in this fight, we are experiencing a new reality where more individuals living with Duchenne and Becker have access to therapies than…

Know More

Agamree (vamorolone) has been recommended for use in the National Health Service (NHS) in England, Wales, and Northern Ireland for treating Duchenne muscular dystrophy (DMD) patients, 4 and older. The recommendation came from the U.K.’s National Institute for Health and Care Excellence (NICE) and follows Agamree’s approval in the U.K. early this year. Agamree, a…

Know More

My wife, Wendy, and I had a wonderful Sunday recently. We rose early and hit the road to Shanksville, Pennsylvania, where we had lived for over 41 years before moving to Pittsburgh to be closer to our kids and grandkids. I’d agreed to play for the 10:30 a.m. service at our former church, the Unity…

Know More

The venture philanthropy organization SOLVE FSHD is offering $10 million in prizes for innovators who are working to develop new treatments for facioscapulohumeral muscular dystrophy (FSHD). “This competition will bring together the brightest minds in medicine, technology, and science, all working toward a cure for this devastating disease,” Eva Chin, executive director of SOLVE FSHD,…

Know More

Last month, families from around the world came together for PPMD Together: Virtual Edition. This unique virtual gathering provided families a space to connect, share their experiences, and find support from one another as they navigate the daily challenges of living with Duchenne and Becker muscular dystrophy. Following on the heels of two successful in-person…

Know More