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 …
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 …
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, …
New technology may inform effective MD gene therapies: Study
Nov 19, 2024 · Gene therapies for MD. Muscular dystrophy refers to a group of diseases that cause progressive muscle weakness and wasting. DMD, the most common type of muscular …
Once weekly corticosteroid may help some with muscular dystrophy
4 days ago · 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 …
Capricor now seeking FDA approval of DMD therapy deramiocel
Oct 10, 2024 · After three years of treatment in the extension study, data showed sustained benefits in heart and arm function compared with the decline seen among untreated patients.. …
Duchenne muscular dystrophy: Exercise and physical therapy
Jun 17, 2024 · A physical therapist knowledgeable about DMD and progressive muscle weakness and other DMD symptoms can create a therapeutic exercise program that safely stretches and …
Gene therapy BB-301 at low dose helping OPMD patients swallow
Oct 15, 2024 · The trial, cleared by U.S. regulators last year, is evaluating BB-301’s safety and its effects on swallowing in OPMD patients.In its Phase 1b part, patients are being treated …
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 …
FDA awards fast track designation to DM1 treatment DYNE-101
2 days ago · Fast track designation follows positive clinical data from ACHIEVE. The ACHIEVE trial is ongoing. A Phase 1/2 clinical study (NCT05481879), ACHIEVE is still recruiting adults …