Boys with Duchenne muscular dystrophy (DMD) may be able to regain lost height and overcome growth stunting by switching from traditional steroids to the newer medication Agamree (vamorolone), a new study suggests. While standard corticosteroids are essential for managing DMD-related inflammation, they are known to halt bone…

Bridgebio Pharma has submitted an application asking the U.S. Food and Drug Administration (FDA) to approve its experimental oral therapy, BBP-418, to treat limb-girdle muscular dystrophy type 2i (LGMD2i). If approved, BBP-418 would become the first available treatment for LGMD2i, also called LGMDR9. In fact, according to…

As parents, we dedicate our lives to raising our children. We work long and hard, striving to meet their needs, teach kindness, encourage a strong work ethic, and foster self-advocacy. Our goal is to help them grow into successful adults. As a mom of many, I can see my and…

SRP-1001 and SRP-1003, Sarepta Therapeutics’ investigational RNA-based therapies for two types of muscular dystrophy, were generally well tolerated in early results, according to data from two Phase 1/2 trials. SRP-1001, for facioscapulohumeral muscular dystrophy type 1 (FSHD1), and SRP-1003, for myotonic dystrophy type 1, showed high…