Perry Shieh

Opto-Electrophysiological Method to Study Human Skeletal Muscle Channelopathies

National Institute of Arthritis and Musculoskeletal and Skin Diseases · R21

PI · $360.4K · University of California Los Angeles

A Phase 1b Study of Adenylosuccinic Acid (ASA-001) for Adenylosuccinate Synthase 1 (ADSS1) Deficient Myopathy.

Principal investigator · Enrolling by invitation · Phase 1

Cure ADSSL1

Phase 3 Study to Evaluate Efficacy of Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS)

Principal investigator · Completed · Phase 3

Catalyst Pharmaceuticals, Inc.

A Phase 2 Study to Evaluate the Dose and Pharmacodynamic Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy

Principal investigator · Completed · Phase 2

Ultragenyx Pharmaceutical Inc

Expanded Access Study Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS),Congenital Myasthenic Syndrome

Principal investigator · Approved for marketing

Catalyst Pharmaceuticals, Inc.