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NCT05992532 2026-02-11

GammaGA: Prevalence of Acid Sphingomyelinase Deficiency Disease (ASMD) and Gaucher Disease in Patients With Monoclonal Gammopathies and/or Multiple Myeloma

Recruiting

210 enrolled

Plasma cell

Gaucher Disease, Multiple Myeloma, Niemann-Pick Diseases, Paraproteinemias, Splenomegaly

NCT03721068 2025-09-09

Study of CAR T-Cells Targeting the GD2 With IL-15+iCaspase9 for Relapsed/Refractory Neuroblastoma or Relapsed/Refractory Osteosarcoma

Phase 1 Recruiting

18 enrolled

CNSSarcoma

Gaucher Disease, Neuroblastoma, Osteosarcoma

NCT06539169 2024-11-14

FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

Recruiting

1,000 enrolled

Classical hematologyOtherPlasma cell

Alzheimer Disease, Amyloidosis, Amyotrophic Lateral Sclerosis, Anemia, Sickle Cell, Cystic Fibrosis, Ehlers-Danlos Syndrome, Gaucher Disease, Glycogen Storage Disease Type II, Huntington Disease, Myasthenia Gravis, Gangliosidosis, GM1, alpha-Thalassemia, beta-Thalassemia, Muscular Dystrophy, Duchenne, Insomnia, Fatal Familial, Rare Diseases