Post-Trial Access to ATH434 for Patients With Multiple System Atrophy

A Study to Explore the Safety and Tolerability of Acthar in Patients With Amyotrophic Lateral Sclerosis

NCT01906658 ·Status: COMPLETED ·Phase: PHASE2

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

NCT01826487 ·Status: COMPLETED ·Phase: PHASE3

Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 Patients

NCT06421831 ·Status: RECRUITING ·Phase: PHASE1/PHASE2

Exenatide Once-weekly as a Treatment for Multiple System Atrophy

NCT04431713 ·Status: COMPLETED ·Phase: PHASE2

A Study of TAK-341 in Treatment of Multiple System Atrophy

NCT05526391 ·Status: COMPLETED ·Phase: PHASE2

Study of Ataluren in Participants With Nonsense Mutation Aniridia

NCT02647359 ·Status: COMPLETED ·Phase: PHASE2

A Study of SGT-212 Gene Therapy in Friedreich's Ataxia

NCT07180355 ·Status: RECRUITING ·Phase: PHASE1

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

NCT01363401 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

Safety Study of XT-150 in Participants With ALS

NCT06704347 ·Status: NOT_YET_RECRUITING ·Phase: PHASE1

An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy

NCT02090959 ·Status: TERMINATED ·Phase: PHASE3

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

NCT05521022 ·Status: RECRUITING ·Phase: PHASE1

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

NCT05337553 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE3

Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies

NCT03837184 ·Status: COMPLETED ·Phase: PHASE3

Autologous Stem Cell Therapy in Patients With Multiple System Atrophy

NCT07465198 ·Status: NOT_YET_RECRUITING ·Phase: PHASE2

Safety and Efficacy Evaluation of GC101 Gene Therapy Via Intrathecal (IT) Injectionin the Treatment of Patients With Type 2 Spinal Muscular Atrophy (SMA) - Phase III

NCT06971094 ·Status: RECRUITING ·Phase: PHASE3

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

NCT02819557 ·Status: COMPLETED ·Phase: PHASE2

A Pivotal Study of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T)

NCT06673056 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE3

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

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

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

NCT01348451 ·Status: UNKNOWN ·Phase: PHASE1

Phase 2 Open-label Extension Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)

NCT06547216 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE2

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

NCT02644668 ·Status: COMPLETED ·Phase: PHASE2

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

NCT01247207 ·Status: COMPLETED ·Phase: PHASE3

Study to Evaluate Multiple Ascending Dose and Multi-Dose of DT-216 in Adult Patients With Friedreich Ataxia

NCT05573698 ·Status: COMPLETED ·Phase: PHASE1

Expanded Access Program (EAP) of Levacetylleucine for Ataxia-Telangiectasia (A-T)

NCT07380165 ·Status: AVAILABLE