Disease | Sponsor | Treatment | Center | Contact | Key Information |
---|---|---|---|---|---|
Metachromatic Leukodystrophy | Takeda/Shire | intrathecal ERT (SHP611), Multicenter | Hospital Universitari Vall d'Hebron, Barcelona, Spain | ||
Adrenoleukodystrophy | Bluebird | Lenti-D, Multicenter | Kremlin Bicêtre, France | Contact | closed for inclusions |
Vanishing White Matter | Amsterdam UMC | Guanabenz, open label, Monocenter | Amsterdam Leukodystrophy Center | Contact | |
Alexander disease | Ionis | Intrathecal antisense oligonucleotide, Multicenter | Amsterdam Leukodystrophy Center | Contact | |
Pelizaeus-Merzbacher disease | Amsterdam UMC | Deferiprone, open label, Monocenter | Amsterdam Leukodystrophy Center | Contact | Not yet started |
PassageBio | intrathecal gene therapy (PBKR03), Multicenter | Amsterdam Leukodystrophy Center | Contact | ||
Adrenoleukodystrophy | Minoryx | MIN-102, Multicenter | Kremlin Bicêtre, France | Contact | |
Metachromatic leukodystrophy | Takeda/Shire | intrathecal ERT (SHP611), Multicenter | Kremlin Bicêtre, France | Contact | Closed for inclusions |
Metachromatic Leukodystrophy | Takeda/Shire | intrathecal ERT (SHP611), Multicenter | University Children's Hospital Tübingen | Contact | |
Metachromatic Leukodystrophy | Takeda/Shire | intrathecal ERT (SHP611), Multicenter | Amsterdam Leukodystrophy Center | Contact | Closed for inclusions |
Adrenoleukodystrophy | SwanBio | AAV-9 gene therapy to stop progression of myelopathy, Multicenter | Amsterdam Leukodystrophy Center | Contact | |
Adrenoleukodystrophy | MGH | Pramipexole, Multicenter | Amsterdam Leukodystrophy Center | Contact | |
Adrenoleukodystrophy | Poxel | PXL770, Multicenter | Amsterdam Leukodystrophy Center | Contact | Not yet started |