Long-Term Effects of Atidarsagene Autotemcel for Metachromatic Leukodystrophy

F Francesca Fumagalli (IRCCS San Raffaele Scientific Institute, Milan) V Valeria Calbi (IRCCS San Raffaele Scientific Institute, Milan) V Vera Gallo (San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan) A Alberto Andrea Zambon (Neurology Unit and Neurophysiology Service, IRCCS San Raffaele Scientific Institute, Milan) S Salvatore Recupero (San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan) F Francesca Ciotti (Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, Milan) M Marina Sarzana (Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, Milan) M Maddalena Fraschini (Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, Milan) S Stefano Scarparo (Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, Milan) F Fabiola De Mattia (San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan) S Simona Miglietta (San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan) C Clelia Pierini (San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan) M Matias Soncini (San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan) F Francesco Morena (Link Campus University, Rome) E Eugenio Montini F Federica Barzaghi (San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan) G Giulia Consiglieri (San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan) F Francesca Ferrua (San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan) M Maddalena Migliavacca (San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan) F Francesca Tucci (San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan) E Elena Sophia Fratini (Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, Milan) A Alessia Ippolito (Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, Milan) P Paolo Silvani (Department of Anesthesia and Critical Care, IRCCS San Raffaele Scientific Institute, Milan) M Maria Rosa Calvi (Department of Anesthesia and Critical Care, IRCCS San Raffaele Scientific Institute, Milan) A Alessandra Clerici (San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan) A Ambra Corti (San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan) M Marcella Facchini (San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan) S Sara Locatelli (San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan) M Mara Sangalli (San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan) S Stefano Zancan (Fondazione Telethon ETS, Milan) F Federica Miotto (Fondazione Telethon ETS, Milan) M Maria Grazia Natali Sora (Neurology Unit and Neurophysiology Service, IRCCS San Raffaele Scientific Institute, Milan) C Cristina Baldoli (Neuroradiology Unit, IRCCS San Raffaele Scientific Institute, Milan) S Sabata Martino (University of Perugia, Perugia, Italy) A Angélica Córdoba-Claros (Orchard Therapeutics (Europe), London) S Sean L. Moro (Orchard Therapeutics North America, Boston) N Nicholas D. Gollop (Orchard Therapeutics (Europe), London) J Jeff Abate (Orchard Therapeutics North America, Boston) M Muska N. Yarzi (Orchard Therapeutics (Europe), London) P Philippa Nutkins (Orchard Therapeutics (Europe), London) A Andrew Shenker (Clinical Consultant, Pennington, NJ) M Mattia Calissano (Orchard Therapeutics (Europe), London) J Jean Brooks (Orchard Therapeutics (Europe), London) A Alan Richardson (Orchard Therapeutics (Europe), London) L Laura Campbell (Orchard Therapeutics (Europe), London) M Massimo Filippi (Neurology Unit and Neurophysiology Service, IRCCS San Raffaele Scientific Institute, Milan) L Luigi Naldini M Maria Pia Cicalese (San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan) F Fabio Ciceri (Vita-Salute San Raffaele University, Milan) M Maria Ester Bernardo (San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan) A Alessandro Aiuti

Article Details

Volume / Issue Vol. 392, Issue 16
Published April 24, 2025
Pages 1609-1620
ISSN 0028-4793
Publisher Massachusetts Medical Society

Journal Info

New England Journal of Medicine

Massachusetts Medical Society

ISSN: 0028-4793 Social Sciences

Authors (51)

F

Francesca Fumagalli

IRCCS San Raffaele Scientific Institute, Milan

V

Valeria Calbi

IRCCS San Raffaele Scientific Institute, Milan

V

Vera Gallo

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan

A

Alberto Andrea Zambon

Neurology Unit and Neurophysiology Service, IRCCS San Raffaele Scientific Institute, Milan

S

Salvatore Recupero

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan

F

Francesca Ciotti

Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, Milan

M

Marina Sarzana

Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, Milan

M

Maddalena Fraschini

Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, Milan

S

Stefano Scarparo

Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, Milan

F

Fabiola De Mattia

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan

S

Simona Miglietta

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan

C

Clelia Pierini

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan

M

Matias Soncini

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan

F

Francesco Morena

Link Campus University, Rome

E

Eugenio Montini

F

Federica Barzaghi

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan

G

Giulia Consiglieri

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan

F

Francesca Ferrua

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan

M

Maddalena Migliavacca

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan

F

Francesca Tucci

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan

E

Elena Sophia Fratini

Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, Milan

A

Alessia Ippolito

Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, Milan

P

Paolo Silvani

Department of Anesthesia and Critical Care, IRCCS San Raffaele Scientific Institute, Milan

M

Maria Rosa Calvi

Department of Anesthesia and Critical Care, IRCCS San Raffaele Scientific Institute, Milan

A

Alessandra Clerici

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan

A

Ambra Corti

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan

M

Marcella Facchini

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan

S

Sara Locatelli

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan

M

Mara Sangalli

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan

S

Stefano Zancan

Fondazione Telethon ETS, Milan

F

Federica Miotto

Fondazione Telethon ETS, Milan

M

Maria Grazia Natali Sora

Neurology Unit and Neurophysiology Service, IRCCS San Raffaele Scientific Institute, Milan

C

Cristina Baldoli

Neuroradiology Unit, IRCCS San Raffaele Scientific Institute, Milan

S

Sabata Martino

University of Perugia, Perugia, Italy

A

Angélica Córdoba-Claros

Orchard Therapeutics (Europe), London

S

Sean L. Moro

Orchard Therapeutics North America, Boston

N

Nicholas D. Gollop

Orchard Therapeutics (Europe), London

J

Jeff Abate

Orchard Therapeutics North America, Boston

M

Muska N. Yarzi

Orchard Therapeutics (Europe), London

P

Philippa Nutkins

Orchard Therapeutics (Europe), London

A

Andrew Shenker

Clinical Consultant, Pennington, NJ

M

Mattia Calissano

Orchard Therapeutics (Europe), London

J

Jean Brooks

Orchard Therapeutics (Europe), London

A

Alan Richardson

Orchard Therapeutics (Europe), London

L

Laura Campbell

Orchard Therapeutics (Europe), London

M

Massimo Filippi

Neurology Unit and Neurophysiology Service, IRCCS San Raffaele Scientific Institute, Milan

L

Luigi Naldini

M

Maria Pia Cicalese

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan

F

Fabio Ciceri

Vita-Salute San Raffaele University, Milan

M

Maria Ester Bernardo

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan

A

Alessandro Aiuti