Pediatrics, Perinatology and Child Health; Neurology (clinical)
Disciplines :
Pediatrics
Author, co-author :
Ramdas, Sithara; MDUK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford, UK, Department of Paediatric Neurology, John Radcliffe Hospital, Oxford, UK
Servais, Laurent ; Université de Liège - ULiège > Département des sciences cliniques
Horton, R.H., Saade, D., Markati, T., et al. A systematic review of adeno-associated virus gene therapies in neurology: the need for consistent safety monitoring of a promising treatment. J. Neurol. Neurosurg. Psychiatry 93 (2022), 1276–1288.
Miller, T.M., Cudkowicz, M.E., Genge, A., et al. Trial of antisense oligonucleotide Tofersen for SOD1 ALS. N. Engl. J. Med. 387 (2022), 1099–1110.
Markati, T., Duis, J., Servais, L., Therapies in preclinical and clinical development for Angelman syndrome. Expet Opin. Invest. Drugs 30 (2021), 709–720.
Moshe-Lilie, O., Riccelli, L.P., Karam, C., Possible recurrent aseptic meningitis associated with nusinersen therapy. Muscle Nerve 62 (2020), E79–E80.
Scheijmans, F.E.V., Cuppen, I., Zwartkruis, M.M., et al. Inflammatory markers in cerebrospinal fluid of paediatric spinal muscular atrophy patients receiving nusinersen treatment. Eur. J. Paediatr. Neurol. 42 (2022), 34–41.
