Dr. Caroline Sevin
Hôpitaux de Paris Institut du Cerveau ICM Paris
Dr. Caroline Sevin is a pediatric neurologist working at Kremlin Bicêtre hospital. She received her MD in Pediatrics and her PhD at the University Paris Descartes, Paris.
She leads the French reference center for leukodystrophies in children at Kremlin Bicêtre hospital, France. She has long–standing interests in neurodegenerative metabolic diseases, including lysosomal, peroxysomal and mitochondrial diseases.
She has a strong background in leukodystrophies, particularly ALD, MLD and Krabbe disease. CS is PI or co-PI of several clinical trials, mostly in ALD and MLD, using innovative therapies (ex vivo and in vivo gene therapy, intrathecal enzyme replacement therapy, small molecules).
Her research activity focuses on developing in vivo/ex vivo gene therapy approaches for leukodystrophies. During her PhD and post-doc course, she has developed an in vivo gene therapy approach for metachromatic leukodystrophy that led to a clinical trial in early onset MLD patients at Kremlin Bicêtre hospital.
Caroline Sevin is leading the « leukodystrophy disease group » at the ERN-RND in association with Dr Nicole Wolf.
She’s member of several DSMBs for gene therapy trials, member of the ASGCT, SSIEM, EAN, EPNS and member of the ELA and VML Scientific committees.