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 - Concepedia

Concepedia

Publication | Open Access

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

DOI Full Paper Access

266

Citations

26

References

2022

Year

Francesca Fumagalli, Valeria Calbi, Maria Grazia Natali Sora, Maria Sessa, Cristina Baldoli, Paola M. V. Rancoita, Francesca Ciotti, Marina Sarzana, Maddalena Fraschini, Alberto A. Zambon,

Abstract

Orchard Therapeutics, Fondazione Telethon, and GlaxoSmithKline.

References

	Year	Citations

Page 1