Product
Osteopetrosis Only Preparative Regimen
1 clinical trial
29 indications
Indication
Mucopolysaccharidosis DisordersIndication
Hurler syndromeIndication
Mucopolysaccharidosis II (MPS II)Indication
Maroteaux Lamy SyndromeIndication
Sly SyndromeIndication
alpha-mannosidosisIndication
FucosidosisIndication
AspartylglucosaminuriaIndication
Glycoprotein Metabolic DisordersIndication
SphingolipidosesIndication
recessive leukodystrophiesIndication
Globoid Cell LeukodystrophyIndication
Metachromatic LeukodystrophyIndication
Niemann-Pick BIndication
Niemann-Pick C Subtype 2Indication
Niemann-Pick diseaseIndication
Peroxisomal DisordersIndication
AdrenoleukodystrophyIndication
Zellweger SyndromeIndication
Neonatal AdrenoleukodystrophyIndication
Infantile Refsum DiseaseIndication
Acyl-CoA Oxidase DeficiencyIndication
D-Bifunctional Enzyme DeficiencyIndication
Multifunctional Enzyme DeficiencyIndication
Alpha-methylacyl-CoA Racmase DeficiencyIndication
OsteopetrosisIndication
Inherited Metabolic DisordersClinical trial
MT2013-31: Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis Following Conditioning With Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATGStatus: Recruiting, Estimated PCD: 2025-07-14