With the first gene therapy for Duchenne muscular dystrophy (DMD) now approved in the U.S. for most people with…
Articles by Marisa Wexler, MS
Four experimental treatments — deramiocel, sevasemten, satralizumab, and tadalafil — all in clinical testing and all aiming to slow the…
In its 30-year history, Parent Project Muscular Dystrophy (PPMD) has helped fund the development of many experimental treatments for…
More than half a dozen exon-skipping therapies for Duchenne muscular dystrophy (DMD) — all seeking to increase production of…
Note: This story was updated Oct. 22, 2024, to remove a reference to the therapy being pulled from the…
Connecting with a supportive community is essential for people affected by muscular dystrophy to maintain both their emotional and physical…
Parent Project Muscular Dystrophy (PPMD) held its first conference in 1994, in Orlando, Florida. A year later, at its…
Capricor Therapeutics will soon meet with the U.S. Food and Drug Administration (FDA) to discuss plans for an application…
The Parent Project Muscular Dystrophy (PPMD) 30th annual conference kicks off today, offering families affected by Duchenne muscular…
The U.S. Food and Drug Administration (FDA) has expanded its approval of Elevidys (delandistrogene moxeparvovec-rokl), authorizing the one-time gene…