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Muscular Dystrophy News
5 天
DYNE-101 is leading to functional gains for DM1 patients in trial
DYNE-101 eased myotonia and led to other functional improvements for people with myotonic dystrophy type 1 (DM1) in a ...
Muscular Dystrophy News
8 天
My original diagnosis was incorrect. Here’s why that’s important.
Columnist Patrick Moeschen was given an incorrect diagnosis of Becker muscular dystrophy, years before learning he actually ...
Muscular Dystrophy News
12 天
Gene-editing therapy for LGMD shows promise in mouse model
A gene-editing therapy may correct a defect in the DYSF gene that drives a form of limb-girdle muscular dystrophy (LGMD), per ...
Muscular Dystrophy News
7 天
GIVI-MPC named FDA orphan drug for treatment of Becker MD
GIVI-MPC, a stem cell therapy for certain types of muscular dystrophy, has been granted orphan drug status for treating ...
Muscular Dystrophy News
14 天
Application finalized for DMD cell therapy CAP-1002, now deramiocel
Capricor's biologics license application for deramiocel (CAP-1002) is draws largely on the Phase 2 HOPE-2 trial and its ...
Muscular Dystrophy News
13 天
How ‘The Remarkable Life of Ibelin’ moved me as a DMD survivor
Columnist Shalom Lim saw elements of his own journey with DMD reflected in the Netflix documentary "The Remarkable Life of ...
Muscular Dystrophy News
4 天
Stuck in limbo with both accessible vans out of commission
An accident and a malfunction render both of columnist Betty Vertin's accessible vans useless, leaving the family without transportation.
Muscular Dystrophy News
6 天
How my FSHD progression leads to out-of-body experiences
Many times over the years since the 1989 car crash that nearly killed me, I’ve been asked if I had any near-death or out-of-body experiences while I hung between life and death. I always try to ...
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