PMLiVE

Novartis’ Itvisma gets FDA approval for spinal muscular atrophy treatment

Novartis’ Itvisma (onasemnogene) has been approved by the US FDA to treat spinal muscular atrophy (SMA) in children aged two years and older, teenagers and adults with a confirmed mutation in the ...
CBC.ca

Family distraught as Manitoba maintains it won't pay for treatment for 30-year-old with degenerative disease

Tears gathered in Jeremy Bray’s eyes as he absorbed the news that his pleas for Manitoba’s government to cover his life-sustaining treatment hadn’t changed the health minister’s mind. Bray, 30, who ...
Business Wire

Sarepta Provides Progress Update for SRP-1003, its Investigational siRNA treatment for Myotonic Dystrophy Type 1

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today shared progress in the Phase 1/2 multiple ascending dose ...
Eagle-Tribune

Sarepta Provides Progress Update for SRP-1003, its Investigational siRNA treatment for Myotonic Dystrophy Type 1

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today shared progress in the Phase 1/2 multiple ascending dose (MAD) clinical study of SRP-1003 ...
Science-Based Medicine

Hope, Evidence, and Hard Questions in Duchenne Muscular Dystrophy

I’ve been reflecting on the disappointing outcomes and the reaction to Sarepta Therapeutics’ recently announced clinical trial results for its Duchenne muscular dystrophy treatments, AMONDYS 45 ...
BioSpace

Satellos Announces Publication in Nature Communications Supporting its Novel Treatment Approach for Duchenne Muscular Dystrophy

Research confirms muscle stem cell dysfunction impairs polarity and impedes production of myogenic progenitors during fetal development Findings solidify AAK1 as a paradigm shifting drug target to ...