Not applicableLooking for participantsNCT03333200What this trial is testingLongitudinal Study of Neurodegenerative DisordersWho this might be right forMLDKrabbe DiseaseALD+24 more University of Pittsburgh 1,500
Early research (Phase 1)Study completedNCT00008450What this trial is testingTotal-Body Irradiation Followed By Cyclosporine and Mycophenolate Mofetil in Treating Patients With Severe Combined Immunodeficiency Undergoing Donor Bone Marrow TransplantWho this might be right forAdenosine Deaminase DeficiencyAutosomal Recessive DisorderImmune System Disorder+4 more Fred Hutchinson Cancer Center 6
Not applicableLooking for participantsNCT06092346What this trial is testingA Natural History Study Seeks to Understand the Clinical, Genomic, Pharmacological, Laboratory, and Dietary Determinates of Pyrimidine and Purine Metabolism DisordersWho this might be right forAMPD3, OMIM*102772, AMP Deaminase DeficiencyAK1, OMIM *103000, Adenylate Kinase DeficiencyAMPD1, OMIM *102770, Myopathy Due to Myoadenylate Deaminase Deficiency+25 more National Human Genome Research Institute (NHGRI)