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Clinical Trials for Muscular Diseases

• Study Evaluating Efficacy and Safety of Froniglutide (PF1801) in Patients With Idiopathic Inflammatory Myopathy
• Add-on Intravenous Immunoglobulins in Early Myositis
• Absorption, Metabolism, Excretion and Absolute Bioavailability of EDG-5506 in Healthy Volunteers
• A Study to Evaluate Vitamin B3 Derivative to Treat Mitochondrial Myopathy
• Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients
• Clinical Characteristics and Mechanism Research of Inhibitors of Janus Kinase in the Treatment of Idiopathic Inflammatory Myopathies
• An Open Label, Long Term Safety Study of REN001 in Primary Mitochondrial Myopathy Patients (Stride Ahead)
• Study to Evaluate the Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease Resulting From Nuclear DNA Mutations (nPMD)
• Clinical Study of MMF in Treatment of IIM-ILD and Its Effect on Peripheral Blood Treg Cells
• A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy (HOPE-3)
• Changes of Motor Function Tests in Congenital Myopathy Subjects Treated With Oral Salbutamol as Compared to no Treatment
• Tamoxifen Therapy for Myotubular Myopathy
• Early Phase Human Drug Trial to Investigate DYN101 in Participants 2 to 17 Years With Centronuclear Myopathies
• Efficacy Confirmation Study of NPC-09
• A Study to Evaluate ASP0367 in Participants With Primary Mitochondrial Myopathy
• A Study of the Efficacy and Safety of 24 Week Treatment With REN001 in Patients With Primary Mitochondrial Myopathy
• Monotherapy IVIG Gamunex-C for HMG-CoA Reductase Auto-Antibody Positive Necrotizing Myopathy Treatment (The MIGHT Trial)
• Low-dose Interleukin-2 in Combination With Standard Therapy on Idiopathic Inflammatory Myopathy
• Multi-Center Study of ManNAc for GNE Myopathy
• A Phase IIa Trial of the Oral JAK 1/2 Inhibitor, Baricitinib, in the Treatment of Adult IIM
• S 48168 (ARM 210) for the Treatment of RYR1-related Myopathies (RYR1-RM)
• Low-dose Interleukin-2 Treatment on Idiopathic Inflammatory Myopathy
• Treating Idiopathic Inflammatory Myopathies Related Reduced Bone Mineral Density With Denosumab or Zoledronic Acid
• Early Phase Human Drug Trial to Investigate Dynamin 101 (DYN101) in Patients ≥ 16 Years With Centronuclear Myopathies
• Safety and Efficacy Study of Zilucoplan in Subjects With Immune-Mediated Necrotizing Myopathy
• A Phase Ia/Ib, SAD and MAD Study of of KL1333 in Healthy Subjects and Patients With Primary Mitochondrial Disease
• A Study of the Safety of REN001 in Patients With Primary Mitochondrial Myopathy
• Electromyographic Study for the Help and Guidance of BoNTA Administration in the Treatment of Chronic Pelvic Floor Pain
• NIRS to Diagnose SAMS
• Comparative Study of Strategies for Management of Duchenne Myopathy (DM)
• Study of Pioglitazone in Sporadic Inclusion Body Myositis
• A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
• Oral Ifetroban in Subjects With Duchenne Muscular Dystrophy
• Sodium Thiosulfate for Treatment of Calcinosis Associated With Juvenile and Adult Dermatomyositis
• Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
• Trial to Evaluate the Efficacy and Safety of Abatacept in Combination With Standard Therapy Compared to Standard Therapy Alone in Improving Disease Activity in Adults With Active Idiopathic Inflammatory Myopathy
• Repeat of: A Study to Evaluate Efficacy and Safety of Sublingual TNX-102 SL Tablet Taken at Bedtime in Patients With Fibromyalgia
• Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)
• A Study to Evaluate the Safety of Aceneuramic Acid Extended Release (Ace-ER; UX001) Tablets in Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy (GNEM) (Also Known as Hereditary Inclusion Body Myopathy [HIBM]) Patients With Severe Ambulatory Imp
• Fatty Acid Oxidation Defects and Insulin Sensitivity
• Pharmacological Treatment of a Rare Genetic Disease: N-acetylcysteine in Myopathy Associated Selenoprotein N-related Myopathy (SEPN1-RM)
• A Study to Evaluate eFFIcacy and Safety of Sublingual TNX-102 SL Tablet Taken at Bedtime in Patients With fibRoMyalgia
• Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)
• Safety, Tolerability, and Efficacy of MTP-131 for the Treatment of Mitochondrial Myopathy
• Antioxidant Therapy in RYR1-Related Congenital Myopathy
• An Open Label Phase 2 Study of ManNAc in Subjects With GNE Myopathy
• RTA 408 Capsules in Patients With Mitochondrial Myopathy - MOTOR
• Therapeutic Effect of Recombinant Human Growth Hormone (rhGH) on the Myopathy of Cystinosis
• Phase 2 Study of EPI-743 in Children With Pearson Syndrome
• An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy
• Treatment of TNNT1-Myopathy With L-Tyrosine.
• The Impact of IVIG Treatment on Critical Illness Polyneuropathy and/or Myopathy in Patients With MOF and SIRS/Sepsis
• An Open Label Phase 2 Extension Study of Higher Dose Sialic Acid-Extended Release (SA-ER) Tablets and Sialic Acid-Immediate Release (SA-IR) Capsules in Patients With Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy
• Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
• Phase I Clinical Trial of ManNAc in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)
• The Effect of Fibrate Therapy in Two Patients With Neutral Lipid Storage Disease With Myopathy (NLSDM)
• A Phase 2 Study to Evaluate the Dose and Pharmacodynamic Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy
• Vitamin D Replacement in Statin-Induced Myopathy
• Safety and Pharmacokinetics of Sialic Acid Tables in Patients With Hereditary Inclusion Body Myopathy (HIBM)
• Evaluation of Patients With Statin Myopathy Using an N of 1 Trial Design
• Pharmacokinetic Study on N-acetylneuraminic Acid
• CoEnzyme Q10 in Statin Myopathy
• Muscle Characteristics Associated With Statin Therapy
• Effect of Bezafibrate on Muscle Metabolism in Patients With Fatty Acid Oxidation Defects
• The Effect of Statin Medications on Muscle Performance (The STOMP Study)
• Effects of Statins on Pro- and Antioxidant Status : Link With Statin-Associated Myopathy.
• Stem Cell Transplantation in Idiopathic Inflammatory Myopathy Diseases
• TOM: Testosterone in Older Men With Sarcopenia
• Differences in Atorvastatin Metabolite Ratios as a Diagnostic Tool in Detecting Atorvastatin Induced Myotoxicity
• Intravenous Immune Globulin to Treat Hereditary Inclusion Body Myopathy
• Is Myopathy Part of Statin Therapy (IMPOSTER-16)
• Association Between Systemic Exposure of Atorvastatin and Metabolites and Atorvastatin-induced Myotoxicity
• Pilot Compassionate Use Study of Thioctic Acid Treatment in Mitochondrial Myopathy