Advocacy groups launch clinical trial to improve gene therapy access

Three advocacy organizations are teaming up to run a clinical trial to test if efgartigimod, an approved treatment for certain autoimmune diseases, might let more people with Duchenne muscular dystrophy (DMD) benefit from gene therapies. CureDuchenne, Muscular Dystrophy Association (MDA), and Parent Project Muscular Dystrophy (PPMD)…

Like many families in the Duchenne muscular dystrophy (DMD) community, we’re preparing to send our three sons with the condition to a weeklong camp for young people with muscular dystrophy. I’ve spent the week preparing everything for Max, 17, Rowen, 14, and Charlie, 12, to attend the…

Stem cells derived from the amniotic membrane of pregnant women after childbirth “could provide therapeutic benefits” for people with Duchenne muscular dystrophy (DMD), according to researchers in Japan. These stem cells, known as human mesenchymal stromal cells, were able to delay DMD progression and preserve muscle function in a…

In the U.S., only a limited number of patients will enter Sarepta Therapeutics‘ ENVISION, a Phase 3 study that’s part of the company’s strategy to gain global approval of SRP-9001 (delandistrogene moxeparvovec) as a gene therapy for Duchenne muscular dystrophy (DMD). These DMD patients will be non-ambulatory, or…

A couple weeks ago, as I was looking through social media, a memory from several years ago appeared on my timeline. I have three sons with Duchenne muscular dystrophy (DMD): Max, 17, Rowen, 14, and Charlie, 12. In May 2019, the boys and I traveled to Los Angeles to…

The LELANTOS-1 Phase 3 clinical trial, assessing pamrevlumab in combination with corticosteroids in people with Duchenne muscular dystrophy (DMD) who are no longer able to walk, did not reach its main goal. FibroGen, the therapy’s developer, announced the top-line results from the trial, whose main objective was to assess changes in upper…

One cozy night last September, my parents and I relaxed in the living room after dinner. My father turned to me and asked for a movie recommendation. It’d been awhile since we’d done this — our cherished family tradition of watching movies together on weekend evenings. In 2019, we lost…

MP1032, MetrioPharm’s oral therapy candidate for Duchenne muscular dystrophy (DMD), has been designated an orphan drug by the U.S. Food and Drug Administration (FDA). A Phase 2 trial testing MP1032 in DMD patients is expected to start next year. “Currently, DMD cannot be cured, but it can be treated,”…

Memorial Day weekend has come and gone, and we’re now officially in summer mode at my house. For our family, that means lots of baseball. I have three sons — Max, 17, Rowen, 14, and Charlie, 12 — with Duchenne muscular dystrophy (DMD). But I also have one son,…

The U.S. Food and Drug Administration (FDA) has placed a clinical hold on an application to launch a Phase 1 study of PGN-EDODM1, an experimental medicine by PepGen for people with myotonic dystrophy type 1 (DM1). A clinical hold is an order issued by the FDA to delay…