Long-Term Effects of Atidarsagene Autotemcel for Metachromatic Leukodystrophy
Among patients with presymptomatic late-infantile or early-juvenile MLD and those with early-symptomatic early-juvenile MLD, the risk of severe motor impairment or death was significantly lower among those who received treatment with arsa-cel than in a natural history cohort that did not receive treatment. (Funded by Orchard Therapeutics and others; ClinicalTrials.gov numbers, NCT01560182 and NCT03392987.).
Authors
- Francesca Ferrua (ORCID: https://orcid.org/0000-0002-5695-4490)
- Massimo Filippi (ORCID: https://orcid.org/0000-0002-5485-0479)
- Mara Sangalli
- Cristina Baldoli (ORCID: https://orcid.org/0000-0003-2697-5132)
- Fabio Ciceri (ORCID: https://orcid.org/0000-0003-0873-0123)
- Matías Soncini (ORCID: https://orcid.org/0000-0001-5973-7515)
- Valeria Calbi (ORCID: https://orcid.org/0000-0003-3514-666X)
- Francesca Ciotti
- Maria Pia Cicalese (ORCID: https://orcid.org/0000-0003-0371-3279)
- Marcella Facchini
- Maddalena Fraschini
- Francesco Morena (ORCID: https://orcid.org/0000-0001-9407-0576)
- Salvatore Marco Recupero
- Elena Sophia Fratini
- Andrew Shenker
- Fabiola De Mattia
- Stefano Scarparo (ORCID: https://orcid.org/0009-0006-8514-141X)
- Maria Grazia Natali Sora (ORCID: https://orcid.org/0000-0002-4298-6619)
- Alessandro Aiuti (ORCID: https://orcid.org/0000-0002-5398-1717)
- Stefano Zancan
- Maddalena Migliavacca (ORCID: https://orcid.org/0000-0001-9051-9663)
- Alberto A. Zambon (ORCID: https://orcid.org/0000-0002-6539-5360)
- Francesca Tucci (ORCID: https://orcid.org/0000-0001-8798-8987)
- Alan E. Richardson
- Ambra Corti
- Marina Sarzana
- Paolo Silvani (ORCID: https://orcid.org/0000-0001-9844-3149)
- Eugenio Montini (ORCID: https://orcid.org/0000-0003-1771-6067)
- Vera Gallo (ORCID: https://orcid.org/0000-0002-7928-0599)
- Simona Miglietta (ORCID: https://orcid.org/0000-0001-7306-0627)
- Giulia Consiglieri (ORCID: https://orcid.org/0000-0003-1750-5745)
- Maria Rosa Calvi (ORCID: https://orcid.org/0000-0003-1039-6291)
- Luigi Naldini (ORCID: https://orcid.org/0000-0002-7835-527X)
- Sean Moro
- Federica Barzaghi (ORCID: https://orcid.org/0000-0003-4498-1712)
- Maria Ester Bernardo (ORCID: https://orcid.org/0000-0002-0099-8021)
- Alessia Ippolito
- Francesca Fumagalli (ORCID: https://orcid.org/0000-0001-7476-4087)
- Muska Yarzi
- Laura Campbell (ORCID: https://orcid.org/0000-0003-2593-8006)
- Sabata Martino (ORCID: https://orcid.org/0000-0002-2740-4561)
- Alessandra Clerici
- Sara M. Locatelli (ORCID: https://orcid.org/0000-0001-6935-872X)
- Jean Brooks
- Nicholas D. Gollop
- Philippa Nutkins
- Clelia Pierini
- Federica Miotto
- Angélica Córdoba-Claros
- Jeff Abate
- Mattia Calissano
Institutions
- Vita-Salute San Raffaele University (IT)
- University of Perugia (IT)
- Mylan (South Africa) (ZA)
- The San Raffaele Telethon Institute for Gene Therapy (IT)
- Istituti di Ricovero e Cura a Carattere Scientifico (IT)
- Fondazione Telethon (IT)
- Istituto di Ricovero e Cura a Carattere Scientifico San Raffaele (IT)
Publication Details
- Journal
- New England Journal of Medicine
- Published
- 2025-04-23
- DOI
- https://doi.org/10.1056/nejmoa2405727
- Citations
- 14
- Primary Topic
- Neutrophil, Myeloperoxidase and Oxidative Mechanisms
- Type
- article
- Field-Weighted Citation Impact
- 10.37
Funders
- GlaxoSmithKline
- Fondazione Telethon