Project Cure Spinal Muscular Atrophy
Project Cure SMA is a collaborative initiative between Families of SMA and clinical investigators designed to help facilitate the rapid translation of promising new therapies to individuals with SMA. The primary goal of Project Cure SMA is to develop safe and well-tolerated clinical protocols to help identify effective therapies for SMA.
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Acid Maltase Deficiency Adult Spinal Muscular Atrophy Amyotrophic Lateral Sclerosis - Blogs by People with Amyotrophic Lateral Sclerosis - Memorials Becker muscular dystrophy Carnitine Deficiency Carnitine Palmityl Transferase Deficiency Central Core Disease Charcot-Marie-Tooth - Blogs by People with Charcot-Marie-Tooth Congenital Muscular Dystrophy Congenital Myasthenic Syndromes Dejerine-Sottas Dermatomyositis Distal Muscular Dystrophy Duchenne's muscular dystrophy - Blogs by People with Duchenne's Emery-Dreifuss Muscular Dystrophy Facioscapulohumeral Muscular Dystrophy Friedreich's Ataxia - People with Friedreich's Ataxia General - Debrancher Enzyme Deficiency Hyperthyroid Myopathy Hypothyroid Myopathy Inclusion Body Myositis Infantile Progressive Spinal Muscular Atrophy Lactate Dehydrogenase Deficiency Lambert-Eaton Myasthenic Syndrome Limb-Girdle Muscular Dystrophy Mitochondrial Myopathy Myasthenia Gravis Myoadenylate Deaminase Deficiency Myotonia Congenita Myotonic Dystrophy Myotubular Myopathy Nemaline Myopathy Oculopharyngeal Muscular Dystrophy Paramyotonia Congenita Periodic Paralysis Phosphofructokinase Deficiency Phosphoglycerate Kinase Deficiency Phosphoglycerate Mutase Deficiency Phosphorylase Deficiency Polymyositis Spinal Bulbar Muscular Atrophy Spinal Muscular Atrophy Spinal Muscular Atrophy Type 2 Spinal Muscular Atrophy Type 3
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