Sevasemten lowers markers of muscle damage in BMD: Trial
Dec 17, 2024 · “This landmark study presents compelling biomarker data and promising signals that suggest the potential for functional stabilization with administration of sevasemten,” Craig …
Adaptive aids for Duchenne muscular dystrophy – Muscular …
Jun 21, 2024 · Assistive technologies. Advancements in technology have been a great help to people living with DMD, from enhancing accessibility with eye-tracking devices, smart …
A year after the approval of Elevidys, it's 'no longer just about...
Aug 22, 2024 · Elevidys became the first and only gene therapy available for DMD patients after the U.S. Food and Drug Administration (FDA) conditionally cleared it in June 2023 for boys, …
Gene-editing therapy for LGMD shows promise in mouse model
Jan 9, 2025 · Gene-editing therapy targets DYSF, whose mutations cause LGMD type 2B/R2. MD refers to a group of disorders characterized by progressively worsening muscle weakness and …
Once weekly corticosteroid may help some with muscular dystrophy
Jan 21, 2025 · Prednisone was given once weekly for six months to 19 people with limb-girdle muscular dystrophy (LGMD), a disease type that primarily affects the hips and shoulders, and …
PPMD 2024: Trials due for DMD gene therapies, cell regeneration
Jul 3, 2024 · To get around this, some gene therapies, such as Elevidys (delandistrogene moxeparvovec-rokl), which was recently authorized in the U.S. most DMD patients ages 4 and …
The link between DMD and gastrointestinal issues – Muscular...
Jul 15, 2024 · Prevalence. Many people living with DMD will have a GI issue at some point. It becomes more common as the disease progresses. Williams says he sees an average of six …
NS Pharma DMD treatment gets FDA rare pediatric disease status
Sep 12, 2024 · NS-050/NCNP-03 is an antisense oligonucleotide, a short strand of genetic material. The therapy skips over exon 50, an approach amenable for about 4% of the DMD …
PPMD 2024: 4 therapies to slow DMD progression now in trials
Jul 4, 2024 · In Duchenne MD, mutations lead to a lack of functional dystrophin, a protein that normally helps protect muscles from damage.That lack of dystrophin causes muscles to …
DYNE-101 is leading to functional gains for DM1 patients in trial
Jan 16, 2025 · The Phase 1/2 ACHIEVE trial (NCT05481879) will now enroll a new group of participants who will all receive the dose that’s so far shown the most promising clinical …