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Recruiting trial = Recruiting trial ; Ongoing trial = Ongoing trial ; Terminated trial = Terminated trial; Funded by an IRDiRC member = ; Member of a ERN =

National clinical trial(s)

ITALY

LOMBARDIA
MILANO

Ongoing trial Funded by an IRDiRC memberINTEGRATION CORE: integration site analysis in a clinical trial of lentiviral vector based hematopoietic stem cell gene therapy for metachromatic leukodystrophy
IRCCS Ospedale San Raffaele
Centro di Genomica, Bioinformatica e Biostatistica - Unità di Genetica Umana e Molecolare

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ITALY

LOMBARDIA
MILANO

Recruiting trial Ongoing trial Funded by an IRDiRC memberA Phase I/II Clinical Trial of Hematopoietic Stem Cell Gene Therapy for the Treatment of Metachromatic Leukodystrophy
Istituto San Raffaele Telethon per la Terapia Genica - TIGET
Unità di Ricerca Clinica

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ITALY

LOMBARDIA
MILANO

Recruiting trial Ongoing trial A Single Arm, Open Label, Clinical Study of Cryopreserved Autologous CD34+ Cells Transduced With Lentiviral Vector Containing Human ARSA cDNA (GSK2696274), for the Treatment of Early Onset Metachromatic Leukodystrophy (MLD)
Istituto San Raffaele Telethon per la Terapia Genica - TIGET
Unità di Ricerca Clinica

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