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…
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…
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…
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,…
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…
Once a week treatment with a corticosteroid was seen to lower some biomarkers of muscle damage and inflammation, helping to improve muscle function in people with some forms of muscular dystrophy, according to a pilot study in the U.S. Prednisone was given once weekly for six months to 19…
I live in rural Nebraska with my husband and our seven children. Three of them — Max, 19; Rowen, 15; and Charlie, 14 — live with Duchenne muscular dystrophy (DMD). People who meet me in person for the first time when I’m with my family are often curious to…
The investigational therapy DYNE-101 worked as expected and led to functional improvements for people with myotonic dystrophy type 1 (DM1) in a clinical trial, according to an update from developer Dyne Therapeutics. The Phase 1/2 ACHIEVE trial (NCT05481879) will now enroll a new group of participants…
Many times over the years since the 1989 car crash that nearly killed me, I’ve been asked if I had any near-death or out-of-body experiences while I hung between life and death. I always try to clarify what people mean by these terms. If we’re talking about my spirit…
Note: This story was updated Jan. 17, 2025, to clarify the company’s comments on gene therapies, which generally deliver a smaller form of dystrophin. The U.S. Food and Drug Administration (FDA) has granted IPS Heart’s GIVI-MPC — a stem cell therapy designed to make new muscle tissue in people…
Get regular updates to your inbox.