12.07.2023
FGCL-3019-093 LELANTOS ONE
A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in Subjects with Non ambulatory Duchenne Muscular Dystrophy (DMD)
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SPIMM-301
A Phase 3 Randomized, Double-Blind, Parallel-Group, PlaceboControlled Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Subjects with Primary Mitochondrial Myopathy Followed by an Open-Label Treatment Extension
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REN001-201
A DOUBLE-BLIND, PLACEBO-CONTROLLED, study to EVALUATE the EFFICACY and safety of 24 weeks treatment with REN001 in patients with primary mitochondrial myopathy (PMM)
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PTC743-NEU-003-FA MOVE-FA
A randomized, parallel-arm, double-blind, placebo-controlled study with open-label extension to assess the efficacy and safety of Vatiquinone for the treatment of Friedreich Ataxia (move-fa)
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PTC743-MIT-001-EP MIT-E
Efficacy and safety study of vatiquinone for the treatment of mitochondrial disease subjects with refractory epilepsy (MIT-E)
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232SM201 NURTURE
An Open-Label Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Subjects With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
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OPBGC&RS 2011-004_CA2012
Prospective open label trial to evaluate the maintenance of the efficacy and the long term safety of EPI-743 in children with Leigh Disease
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ION373-CS1 IONIS
A Phase 1-3, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION373 in Patients with Alexander Disease
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DSC/14/2357/51
Open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD patients who have been previously treated in one of the GIVINOSTAT studies
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