PHASE I/II CLINICAL TRIAL OF HEMATOPOIETIC STEM CELL GENE THERAPY FOR THE TREATMENT OF METACHROMATIC LEUKODYSTROPHY
- 13 Anni 2010/2023
- 5.179.139€ Totale Fondi
Descrizione non disponibile
Pubblicazioni Scientifiche
- ENDOCRINE REVIEWS
The Changing Face of Adrenoleukodystrophy
- JOURNAL OF INHERITED METABOLIC DISEASE
Metachromatic leukodystrophy: A single-center longitudinal study of 45 patients
- LANCET
Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial
