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) A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients LYSO-PROVE – Determine the Prognostic Value of Lyso-Gb1 for Monitoring the Progress of Gaucher Disease Natural History Study of Children With Metachromatic Leukodystrophy