FDA has requested the HOPE-3 clinical study report (CSR) as part of the BLA review processCompany expects to submit updates ...
Zeleciment basivarsen (z-basivarsen) demonstrated sustained functional improvement across multiple clinical measures in the ongoing ACHIEVE clinical trial - ...
Researchers from Carnegie Mellon University have discovered a way to target RNA that could lead to new treatment options for ...
Designation further supports (Z)-Endoxifen program into rare pediatric neuromuscular disease along with previously received Rare Pediatric Disease Designation SEATTLE ...
Endoxifen program into rare pediatric neuromuscular disease along with previously received Rare Pediatric Disease Designation SEATTLE, /PRNewswire/ -- Atossa Therapeutics, Inc. (Nasdaq: ATOS) ("Atossa ...
In boys with DMD, executive skills such as self-control begin to lag around age 8, but show signs of partial catch-up by 14, ...
Commercial launch in Switzerland anticipated in H2 2026Pratteln, Switzerland, January 15, 2026 – Santhera Pharmaceuticals (SIX: SANN) announces ...
Solid Biosciences (SLDB) stock rises as a Phase 1/2 trial for its lead asset SGT-003 in Duchenne muscular dystrophy reaches ...
Jefferies analysts forecast a $1 billion market opportunity for each of Sarepta’s siRNA programs for facioscapulohumeral muscular dystrophy and myotonic dystrophy type 1.
A stretch of viral DNA in the mouse genome gives cells in early-stage embryos the potential to become almost any cell type in ...
Vamorolone is approved and marketed as AGAMREE® for the treatment of Duchenne Muscular Dystrophy (DMD) in the US, European Union, UK and ChinaNew ...
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