UCSD Altman Clinical and Translational Research Institute

La Jolla, California 92093

1 studies enrolling now · 7 studies all time

What they study most

Alpha1-Antitrypsin Deficiency, Amyotrophic Lateral Sclerosis, DM1, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, Multiple Sclerosis, Muscular Diseases, Muscular Disorders, Atrophic, Muscular Dystrophies, Muscular Dystrophy, Duchenne, Myotonic Dystrophy, Myotonic Dystrophy Type 1 (DM1)

Studies at this site

• Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1 — Recruiting now
• A Study to Evaluate the Safety, Tolerability, Cellular Kinetics, and Pharmacodynamics of P-CD19CD20-ALLO1 in Participants With Multiple Sclerosis — Running, not enrolling
• A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy — Running, not enrolling
• An Extension Study to Learn About the Long-Term Safety of Fazirsiran and if Fazirsiran Can Help People With Alpha-1 Antitrypsin Liver Disease — Running, not enrolling
• A Safety and Biomarker Study of ALZT-OP1a in Subjects With Mild-Moderate ALS Disease — Stopped early
• A Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of EDP-305 in Subjects With Non-Alcoholic Steatohepatitis — Completed
• Long-Term Safety Of PF-00547659 In Ulcerative Colitis — Completed