A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation

Study of the Efficacy and Safety of Nicotinamide in Patients With Friedreich Ataxia

NCT03761511 ·Status: WITHDRAWN ·Phase: PHASE2

Safety and Tolerability Study of KNS-760704 in Amyotrophic Lateral Sclerosis (ALS)

NCT00647296 ·Status: COMPLETED ·Phase: PHASE2

A Multiple Ascending Dose Study of DT-216P2 in Patients With Friedreich's Ataxia

NCT06874010 ·Status: RECRUITING ·Phase: PHASE1/PHASE2

Efficacy and Safety of Acoramidis (AG10) in Subjects with Transthyretin Amyloid Polyneurophathy (ATTRibute-PN)

NCT04882735 ·Status: WITHDRAWN ·Phase: PHASE3

Efficacy of Epoetin Alfa in Patients With Friedreich's Ataxia

NCT00631202 ·Status: COMPLETED ·Phase: PHASE2

A Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects With Friedreich's Ataxia

NCT04102501 ·Status: COMPLETED ·Phase: PHASE3

Open-Label, Safety and Tolerability Extension Study of KNS-760704 in Amyotrophic Lateral Sclerosis (ALS) (CL211)

NCT00931944 ·Status: COMPLETED ·Phase: PHASE2

A Clinical Trial to Learn About the Effects of VHB937 in People With Amyotrophic Lateral Sclerosis (ALS)

NCT06643481 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE2

A First in Human Study of RT001 in Patients With Friedreich's Ataxia

NCT02445794 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

A Study to Evaluate the Efficacy and Safety of Reldesemtiv in Patients With Amyotrophic Lateral Sclerosis (ALS)

NCT04944784 ·Status: TERMINATED ·Phase: PHASE3

Study to Investigate the Efficacy and Safety of FAB122 (Daily Oral Edaravone) in Patients With Amyotrophic Lateral Sclerosis

NCT05178810 ·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

A Study to Evaluate, Safety, Tolerability, Pharmacodynamic (PD) Markers and Pharmacokinetics (PK) of AP-101 in Participants With Amyotrophic Lateral Sclerosis (ALS)

NCT05039099 ·Status: COMPLETED ·Phase: PHASE2

Study to Evaluate DT-216 in Adult Patients With Friedreich Ataxia

NCT05285540 ·Status: COMPLETED ·Phase: PHASE1

Study to Evaluate DNL747 in Subjects With Amyotrophic Lateral Sclerosis

NCT03757351 ·Status: TERMINATED ·Phase: PHASE1

Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam

NCT05156320 ·Status: COMPLETED ·Phase: PHASE3

A Study of NP001 in Subjects With Amyotrophic Lateral Sclerosis (ALS)

NCT01281631 ·Status: COMPLETED ·Phase: PHASE2

Dimethyl Fumarate in Adrenomyeloneuropathy

NCT06513533 ·Status: RECRUITING ·Phase: PHASE2/PHASE3

Safety and Pharmacology Study of VP 20629 in Adults With Friedreich's Ataxia

NCT01898884 ·Status: COMPLETED ·Phase: PHASE1

Clinical Study to Evaluate the Efficacy and Safety of FB1006 in the Treatment of ALS Patients

NCT05923905 ·Status: RECRUITING ·Phase: PHASE4

A Study Evaluating the Safety and Tolerability of QRL-201 in ALS

NCT05633459 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1

A Study to Assess the Long-Term Safety and Efficacy of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Patients With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

NCT05071300 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE3

Study of Ataluren in Participants With Nonsense Mutation Aniridia

NCT02647359 ·Status: COMPLETED ·Phase: PHASE2

A Phase II Multi-centre, Extension Study to Investigate the Long Term Safety of ONO-2506PO in Patients Diagnosed With Amyotrophic Lateral Sclerosis (ALS)

NCT00694941 ·Status: COMPLETED ·Phase: PHASE2

R(+) Pramipexole in Early Amyotrophic Lateral Sclerosis

NCT00140218 ·Status: COMPLETED ·Phase: PHASE1/PHASE2