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Human Gene Therapy|February 15, 2014
Intracerebral administration of adeno-associated viral vector serotype rh.10 carrying human SGSH and SUMF1 cDNAs in children with mucopolysaccharidosis type IIIA disease: results of a phase I/II trialMarc Tardieu, Michel Zérah, Béatrice Husson, et al.
Movement Disorders : Official Journal of the Movement Disorder Society|May 23, 2018
Deep brain stimulation treated dystonia-trajectory via status dystonicusElodie Nerrant, Victoria Gonzalez, Christophe Milesi, et al.
The Lancet. Neurology|July 18, 2017
Intracerebral gene therapy in children with mucopolysaccharidosis type IIIB syndrome: an uncontrolled phase 1/2 clinical trialMarc Tardieu, Michel Zérah, Marie-Lise Gougeon, et al.
Human Gene Therapy. Clinical Development|March 12, 2015
Intracerebral Gene Therapy Using AAVrh.10-hARSA Recombinant Vector to Treat Patients with Early-Onset Forms of Metachromatic Leukodystrophy: Preclinical Feasibility and Safety Assessments in Nonhuman PrimatesMichel Zerah, Françoise Piguet, Marie-Anne Colle, et al.
Journal of Inherited Metabolic Disease|July 4, 2023
Gene therapy for aromatic L-amino acid decarboxylase deficiency: Requirements for safe application and knowledge-generating follow-upAgathe Roubertie, Thomas Opladen, Heiko Brennenstuhl, et al.
Plos One|March 6, 2012
Mesenchymal transition and PDGFRA amplification/mutation are key distinct oncogenic events in pediatric diffuse intrinsic pontine gliomasStephanie Puget, Cathy Philippe, Dorine A Bax, et al.
Neuro-Oncology|April 9, 2020
Role of neoadjuvant chemotherapy in metastatic medulloblastoma: a comparative study in 92 childrenLéa Guerrini-Rousseau, Rachid Abbas, Sophie Huybrechts, et al.
Journal of Neuropathology and Experimental Neurology|January 18, 2024
"Hemispheric pilocytic astrocytoma" revisited: A comprehensive clinicopathological and molecular series emphasizing their overlap with other glioneuronal tumorsCassandra Mariet, Jacques Grill, Yassine Ajlil, et al.
Journal of Neurosurgery. Pediatrics|August 2, 2022
Chiari malformation type I surgery in children: French multicenter 10-year cohortEdouard Mazerand, Sandro Benichi, Maxime Taverne, et al.
Journal of the Neurological Sciences|August 15, 2018
Deep brain stimulation is effective in pediatric patients with GNAO1 associated severe hyperkinesiaAnne Koy, Sebahattin Cirak, Victoria Gonzalez, et al.
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