-
MDA honors researcher, patient advocate with 2025 Legacy Awards
The Muscular Dystrophy Association (MDA) has announced its 2025 Legacy Awards, and will honor two individuals who have made pioneering strides toward improving life for people affected by muscular dystrophy. The 2025 MDA Legacy Award for Achievement in Clinical Research will be given to Katherine Mathews, MD, a researcher who has studied muscular dystrophy for…
-
How a Singapore ‘Artist’s Way’ program is rekindling my creativity
Rediscovering my creative self has been one of the most fulfilling yet challenging parts of my journey with Duchenne muscular dystrophy (DMD). A few weeks ago, I started an exciting adventure with Wildly Creative, a 14-week arts and wellness program run by the Singapore theater company Wild Rice. Led by Edith Podesta, an Australian director…
-
Another fall left me feeling like an upside-down turtle
About a month ago, I came home from playing with my church’s music group at a nursing facility. We played a doubleheader, doing a set in the memory care wing, then another in the personal care area. Because of my facioscapulohumeral muscular dystrophy (FSHD), I’ve stopped doing back-to-back performances, aside from a couple times a…
-
Boys with DMD given Elevidys two years ago still showing motor gains
Two years after receiving the gene therapy Elevidys (delandistrogene moxeparvovec-rokl), motor function continues to improve in boys with Duchenne muscular dystrophy (DMD) who entered a global clinical trial able to walk. That’s according to top-line findings from part two of the Phase 3 EMBARK trial (NCT05096221), which evaluated Sarepta Therapeutics‘ approved gene therapy in more…
-
Sarepta’s EMBARK study data shows continuing benefits to ambulatory individuals treated with their microdystrophin gene therapy, Elevidys
Sarepta Therapeutics shared positive topline results from Part 2 of the EMBARK study, showing that treatment with the microdystrophin gene therapy, Elevidys, is associated with sustained benefits and disease stabilization in ambulatory individuals. EMBARK is a Phase 3 study of Elevidys (in individuals aged 4-7 years) with a crossover design, in which individuals treated with…
-
Sarepta Therapeutics Announces Results from Part 2 of EMBARK Study of ELEVIDYS
Sarepta Therapeutics, Inc. has announced positive topline results from Part 2 of EMBARK, the company’s Phase 3 clinical study of ELEVIDYS. ELEVIDYS is a micro-dystrophin gene therapy used for the treatment of individuals aged four years and older with Duchenne. Results show that crossover-treated patients who received a placebo for 52 weeks in Part 1…
-
Update from PPMD’s 2025 Duchenne Healthcare Professionals Summit
PPMD recently convened over 360 healthcare professionals, researchers, and industry partners, including representatives from the FDA, NIH, CDC, and international representatives in one room to discuss the latest updates in dystrophinopathy care and research at the 2025 Duchenne Healthcare Professionals Summit. Key topics of the meeting included: Lessons Learned in Gene Therapy The Summit kicked…
-
FDA awards fast track designation to DM1 treatment DYNE-101
The U.S. Food and Drug Administration (FDA) has granted fast track designation to DYNE-101, a treatment candidate for myotonic dystrophy type 1 (DM1) — in which disease symptoms begin during adulthood — that’s now being tested in a clinical trial involving DM1 patients. This status is awarded by the FDA to experimental therapies that have…
-
What success looks like for me as a DMD parent
Parenting is the most challenging job I’ve ever had. When my role as a parent expanded into caregiving for three of my children, challenging didn’t even begin to describe the path that lay in front of us. I share seven children with my husband, Jason: Lexi, 23, Max, 19, Chance, 17, Rowen, 16, Charlie, 14,…
-
Avidity plans to submit for FDA Accelerated Approval for Duchenne exon 44 skipping at the end of 2025; Open-label extension trial is still recruiting
Amenable to skipping exon 44 Avidity Biosciences, which received early funding from CureDuchenne, plans to submit for US FDA Accelerated Approval at the end of 2025 for Delpacibart zotadirsen (del-zota), an exon-skipping experimental therapeutic for individuals with Duchenne amenable to skipping exon 44. Topline data from their EXPLORE44 trial will be released in Q1 of…
