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Givinostat Slows Duchenne Muscular Dystrophy Decline

Trial details the results that led to recent FDA approval ....

Eugenio Mercuri , Givinostat Duvyzat , Janbernd Kirschner , Sophie Putka , University Of Freiburg , Universita Cattolica Del Sacro Cuore , North Star Ambulatory Assessment , Collaborative Trajectory Analysis Project , Wall Street Journal , Business Insider , Cannabis Wire , Medpage Today ,

Positive Phase 2 Data for SRP-5051 in Duchenne Muscular Dystrophy

The Momentum trial from Sarepta Therapeutics is investigating SRP-5051 (vesleteplirsen) for use among male patients aged 8 to 21 years who have Duchenne muscular dystrophy amenable to exon 51 skipping.
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Eugenio Mercuri , Catholic University , Sarepta Therapeutics , Neuromuscular Unit , Duchenne Muscular , Accessed February , Duchenne Muscular Dystrophy , Srp 5051 , Momentum Trial , Dystrophin Expression ,

Italfarmaco Group Announces Positive Topline Data from Phase 3 Trial Showing Beneficial Effect of Givinostat in Patients with Duchenne Muscular Dystrophy

Italfarmaco Group Announces Positive Topline Data from Phase 3 Trial Showing Beneficial Effect of Givinostat in Patients with Duchenne Muscular Dystrophy
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San Diego , United States , University Of California Davis , Craig Mcdonald , Paolo Bettica , Eugenio Mercuri , Laura Mittmann , Jacob Verghese , Santa Lucia Foundation , Drug Administration , Department Of Pediatrics , Italfarmaco Group , Trophic Communications , Catholic University , Duchenne Muscular Dystrophy , Chief Medical Officer , Parent Project Muscular Dystrophy , Rare Pediatric Disease , Orphan Drug Designation , Fast Track , Clinical Results , Secondary Endpoints , North Star Ambulatory Assessment , Magnetic Resonance Spectroscopy , Adverse Events , Paediatric Neurology ,

Repeated intravenous cardiosphere-derived cell therapy in late-stage Duchenne muscular dystrophy (HOPE-2): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial

Repeated intravenous cardiosphere-derived cell therapy in late-stage Duchenne muscular dystrophy (HOPE-2): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial
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Beverly Hills , United States , United Kingdom , City Of , Veena Ramanna Capricor , Epirium Bio , Capricor Tharapeutics , J Baek Sacramento , Sam Dickson , Mallinckrodt Pharmaceuticals , Rehabilitation Research , Atom International , Mallinkrodt Pharmaceuticals , Therapy Solutions , National Institute On Disability , Logan Kowallis Pentara Corporation , Bristol Myers Squibb , Muscular Dystrophy Association , Vertex Pharmaceuticals , Santhera Pharmaceuticals , Eugenio Mercuri Catholic University , Steering Committee , Study Group , Pentara Corporation , Us Department Of Defense , Institute Of Child Health ,

Risdiplam Shows Benefit in SMA Type 2, Type 3 | Physician's Weekly

Risdiplam Shows Benefit in SMA Type 2, Type 3 | Physician's Weekly
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Paul Smyth , Valeria Sansone , Eugenio Mercuri , Avexis Novartis , University Of Milan , Catholic University , Motor Function Measure , Contributing Writer , Scholar Rock ,