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…
News
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…
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…
Sarepta Therapeutics plans to ask the U.S. Food and Drug Administration (FDA) to grant full approval for Amondys 45 (casimersen) and Vyondys 53 (golodirsen), two exon-skipping therapies designed to treat certain people with Duchenne muscular dystrophy (DMD). The company said it met with the FDA to…
Muscular Dystrophy News Today is wrapping up coverage of the 2026 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, which ran from March 8-11 in Orlando, Florida, and online. This year, more than 2,400 attendees from 40 countries gathered in person and virtually to discuss recent advances in neuromuscular…
Dyne Therapeutics is advancing its investigational exon-skipping therapy zeleciment rostudirsen (z-rostudirsen), formerly known as DYNE-251, toward regulatory approval after trial data showed early signs of benefit across multiple disease measures in boys with Duchenne muscular dystrophy (DMD). The most recent data from the Phase 1/2 DELIVER clinical trial…
In 2018, when Nell Choi was 9 years old, she began experiencing symptoms that led to hospitalization and a diagnosis of neuromyelitis optica spectrum disorder (NMOSD), type positive. A month later, when Nell came home, she was weak, in a wheelchair, and had lost 20 pounds. To…
Boys with Duchenne muscular dystrophy (DMD) who received Sarepta Therapeutics’ gene therapy Elevidys (delandistrogene moxeparvovec-rokl) in a clinical trial continue to show signs of slowed disease progression relative to DMD’s natural course, along with caregiver-reported improvements in everyday living. That’s according to data from the Phase…
The latest Phase 3 trial results continue showing that deramiocel, an investigational cell therapy for heart disease related to Duchenne muscular dystrophy (DMD), significantly slows the progression of arm and heart damage in boys and men with DMD. Based on the positive Phase 3 results, Capricor Therapeutics has…
Myotonic dystrophy type 1 (DM1) patients in a Phase 1/2 clinical trial saw gains in motor function and cognitive measures after receiving Dyne Therapeutics’ zeleciment basivarsen (z-basivarsen), previously known as DYNE-101, supporting the recent initiation of a Phase 3 study. Findings from the Phase 1/2 ACHIEVE trial…
Recent Posts
- Making time for self-care is vital in DMD caregiving
- Agamree could reverse growth stunting in boys with Duchenne
- Bridgebio asks FDA to approve potential 1st treatment for LGMD2i
- I’m grateful for meaningful conversations with my adult children
- Early trial data support RNA therapies for two muscular dystrophy types