| 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 |
| Krabbe disease | 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 |
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ERN-RND | European Reference Network on Rare Neurological Diseases
for rare or low prevalence complex diseases