SPL Insufficiency Syndrome (SPLIS): a SPLIS Observational Study and Patient Registry (International)

Sphingolipidosis

SPL Insufficiency Syndrome (SPLIS): a SPLIS Observational Study and Patient Registry (International) Compassionate Use Program for Olipudase Alfa Enzyme Replacement Therapy for Patients With Chronic Acid Sphingomyelinase Deficiency (ASMD) Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency Natural History Study of Children With Metachromatic Leukodystrophy LYSO-PROVE – Determine the Prognostic Value of Lyso-Gb1 for Monitoring the Progress of Gaucher Disease