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Clinical Trials for Neuromuscular Diseases

• Treatment of Charcot-Marie-Tooth Disease, Axonal, Type 2S (CMT2S) in an Individual Patient
• An Open-Label Extension Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 (FREEDOM-OLE)
• Safety of Intrathecal Riluzole in Patients With Amyotrophic Lateral Sclerosis
• A Study of Long-term Safety and Efficacy of VX-670 in Participants With Myotonic Dystrophy Type I
• A Study to Investigate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AZD5492 in Adult Participants With Systemic Lupus Erythematosus or Idiopathic Inflammatory Myopathies.
• A Study of PGN-EDO51 or Placebo in People With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment
• Efficacy and Safety of Efgartigimod Sequential Therapy with Telitacicept in Generalized Myasthenia Gravis
• A Phase 3 Study of NTLA-2001 in ATTRv-PN
• Safety and Efficacy Study on AFA-281 for the Treatment of Low Back Pain
• An IIT Clinical Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of a Single Intrathecal Injection of SNUG01 in Patients with Amyotrophic Lateral Sclerosis
• Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1
• Absorption, Metabolism, Excretion and Absolute Bioavailability of EDG-5506 in Healthy Volunteers
• Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab-ONYX
• Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients
• A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy
• Phase 3 Active Treatment Trial to Evaluate the Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Who Are Being Treated With Nusinersen or Risdiplam
• 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
• Study of Pioglitazone in Sporadic Inclusion Body Myositis
• A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
• Assessing Air Travel Safety in Neuromuscular Dystrophy: Standard Versus Prolonged High Altitude Simulation Tests
• Treatment Effect of Edaravone in Patients With Amyotrophic Lateral Sclerosis (ALS)
• Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
• AMX0035 in Patients With Amyotrophic Lateral Sclerosis (ALS)
• Repeat of: A Study to Evaluate Efficacy and Safety of Sublingual TNX-102 SL Tablet Taken at Bedtime in Patients With Fibromyalgia
• A Study to Evaluate eFFIcacy and Safety of Sublingual TNX-102 SL Tablet Taken at Bedtime in Patients With fibRoMyalgia
• Antioxidant Therapy in RYR1-Related Congenital Myopathy
• An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy
• Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
• Safety and Efficacy Study of A0001 in Patients With the A3243G Mitochondrial DNA Point Mutation
• Metformin in Children With Motor Deficit
• A Pilot Study To Evaluate the Effect of Retrovir (Zidovudine: AZT) in the Treatment of Human Immunodeficiency Virus (HIV) Associated Dementia and Neuromuscular Diseases