When did I stop being a fun mom for my children?

“I didn’t think you’d jump with us,” said my daughter Mary, 9, breathing heavily as she bounced from trampoline to trampoline. It’s true; I’d never taken her or her little sister Callie, 2, to the trampoline park before. But we do fun things, I thought. After thinking some more, however,…

Parent Project Muscular Dystrophy (PPMD) held its first conference in 1994, in Orlando, Florida. A year later, at its meeting in Pittsburgh, fewer than two dozen people were in attendance, just a small group of parents of children with Duchenne muscular dystrophy (DMD) and a few scientists interested…

Capricor Therapeutics will soon meet with the U.S. Food and Drug Administration (FDA) to discuss plans for an application seeking approval of the company’s CAP-1002 cell therapy, now named deramiocel, as a treatment for Duchenne muscular dystrophy (DMD). The FDA has scheduled the pre-biologics license application (BLA)…

The Parent Project Muscular Dystrophy (PPMD) 30th annual conference kicks off today, offering families affected by Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD) the chance to “celebrate three decades of progress, community, and resilience.” The PPMD conference, which runs through Saturday, is taking place…

Last week, my wife, Wendy, and I finally got around to starting the process of purchasing a new mattress. We realized when we moved to Pittsburgh in January 2023 that we needed to replace our mattress, which has served us well for many more years than the sleep experts…

A Phase 3 trial testing an experimental therapy for limb-girdle muscular dystrophy type 2i (LGMD2i) has surpassed patient enrollment for a planned interim analysis, the therapy’s developer BridgeBio Pharma has announced. The Phase 3 FORTIFY trial (NCT05775848) is evaluating the long-term safety and efficacy of BBP-418 in…

Recently, our family had terrific reasons to celebrate. I have three sons with Duchenne muscular dystrophy (DMD): Max, 18, Rowen, 15, and Charlie, 13. They are doing things we never imagined they’d do when they were young. Max, who recently graduated from high school, was in “The SpongeBob…

The U.S. Food and Drug Administration (FDA) has expanded its approval of Elevidys (delandistrogene moxeparvovec-rokl), authorizing the one-time gene therapy for individuals with Duchenne muscular dystrophy (DMD) ages 4 and older regardless of their ability to walk. Previously, the treatment had been approved for DMD children ages 4…

AOC 1020, an investigational treatment by Avidity Biosciences that’s now called delpacibart braxlosiran, or del-brax, helped adults with facioscapulohumeral muscular dystrophy (FSHD) grow stronger muscles and extend their arms farther than they could four months earlier. These are early data from FORTITUDE (NCT05747924), a three-part Phase 1/2…

The gene therapy developed by Pfizer called fordadistrogene movaparvovec failed to significantly improve motor function in boys with Duchenne muscular dystrophy (DMD) who are able to walk and taking part in a Phase 3 study. The company says it will evaluate the next steps for the fordadistrogene…