Study of ATH434 in Participants with Multiple System Atrophy

Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Participants With Limb Girdle and Facioscapulohumeral Muscular Dystrophy (FSHD)

NCT02836418 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

Safety, Tolerability, Pharmacokinetics, and Biological Activity of ATYR1940 in Adult Participants With Muscular Dystrophy

NCT02239224 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy

NCT02819557 ·Status: COMPLETED ·Phase: PHASE2

A Phase 2 Study of YA-101 in Patients With Multiple System Atrophy

NCT06848231 ·Status: RECRUITING ·Phase: PHASE2

A Study of Lu AF82422 in Participants With Multiple System Atrophy

NCT05104476 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE2

Randomized Double-Blind Placebo-Controlled Adaptive Design Trial Of Intrathecally Administered Autologous Mesenchymal Stem Cells In Multiple System Atrophy

NCT05167721 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE2

Study of AT-02 in Healthy Volunteers and Subjects With Systemic Amyloidosis

NCT05521022 ·Status: RECRUITING ·Phase: PHASE1

Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy

NCT05794139 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE2

Efficacy and Safety Study of MCI-186 for Treatment of Amyotrophic Lateral Sclerosis (ALS) Who Met Severity Classification III

NCT00415519 ·Status: COMPLETED ·Phase: PHASE3

A Phase 2 Study of ONO-2808 in Patients With Multiple System Atrophy

NCT05923866 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE2

Dose Escalation and Safety Study of Human Spinal Cord Derived Neural Stem Cell Transplantation for the Treatment of Amyotrophic Lateral Sclerosis

NCT01730716 ·Status: UNKNOWN ·Phase: PHASE2

Safety, Tolerability, and Exploratory Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants With SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS)

NCT06100276 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1/PHASE2

Study of ATX-01 in Participants With DM1

NCT06300307 ·Status: RECRUITING ·Phase: PHASE1/PHASE2

Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

NCT01826487 ·Status: COMPLETED ·Phase: PHASE3

A Study of CK-2127107 in Patients With Spinal Muscular Atrophy

NCT02644668 ·Status: COMPLETED ·Phase: PHASE2

A Study to Evaluate Efficacy, Safety and Tolerability of CK-2127107 in Patients With Amyotrophic Lateral Sclerosis (ALS)

NCT03160898 ·Status: COMPLETED ·Phase: PHASE2

AMX0114 in Adult Participants With Amyotrophic Lateral Sclerosis

NCT06665165 ·Status: RECRUITING ·Phase: PHASE1

A Safety and Efficacy Study of ARGX-119 in Adult Patients With Amyotrophic Lateral Sclerosis (ALS)

NCT06441682 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE2

A Study of LY4256984 in Participants With Sporadic Amyotrophic Lateral Sclerosis

NCT07100119 ·Status: RECRUITING ·Phase: PHASE1

Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)

NCT01247207 ·Status: COMPLETED ·Phase: PHASE3

A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy

NCT05337553 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE3

A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren

NCT03796637 ·Status: COMPLETED ·Phase: PHASE2

A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients With Spinal Muscular Atrophy (SMA)

NCT02628743 ·Status: COMPLETED ·Phase: PHASE2

Safety and Efficacy Study of Autologous Bone Marrow Derived Stem Cell Treatment in Amyotrophic Lateral Sclerosis

NCT01363401 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

Human Spinal Cord Derived Neural Stem Cell Transplantation for the Treatment of Amyotrophic Lateral Sclerosis

NCT01348451 ·Status: UNKNOWN ·Phase: PHASE1