TrialLancet (London, England)2022

Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access.

Francesca Fumagalli et al.PubMed ↗Full text ↗Publisher ↗

No numbers read from the abstract.