Combination disease-modifying treatment in spinal muscular atrophy: A proposed classification.

Autor: Proud CM; Children's Hospital of The King's Daughters, Norfolk, Virginia, USA., Mercuri E; Department of Paediatric Neurology and Nemo Clinical Centre, Catholic University, Rome, Italy., Finkel RS; Center for Experimental Neurotherapeutics, St. Jude Children's Research Hospital, Memphis, Tennessee, USA., Kirschner J; Department of Neuropediatrics and Muscle Disorders, Medical Center University of Freiburg, Faculty of Medicine, Freiburg, Germany., De Vivo DC; Departments of Neurology and Pediatrics, Columbia University Irving Medical Center, New York, New York, USA., Muntoni F; The Dubowitz Neuromuscular Centre, University College London, Great Ormond Street Institute of Child Health & Great Ormond Street Hospital, London, UK.; National Institute of Health Research, Great Ormond Street Hospital Biomedical Research Centre, London, UK., Saito K; Institute of Medical Genetics, Tokyo Women's Medical University, Tokyo, Japan., Tizzano EF; Department of Clinical and Molecular Genetics, Hospital Vall d'Hebron, Barcelona, Spain., Desguerre I; Hôpital Necker Enfants Malades, APHP, Paris, France., Quijano-Roy S; Garches Neuromuscular Reference Center (GNMH), APHP Raymond Poincare University Hospital (UVSQ Paris Saclay), Garches, France., Benguerba K; Novartis Gene Therapies Switzerland GmbH, Rotkreuz, Switzerland., Raju D; Novartis Gene Therapies, Inc, Bannockburn, Illinois, USA., Faulkner E; Novartis Gene Therapies, Inc, Bannockburn, Illinois, USA.; Institute for Precision and Individualized Therapy, Eshelman School of Pharmacy, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, USA.; Genomics, Biotech and Emerging Medical Technology Institute, National Association of Managed Care Physicians, Richmond, Virginia, USA., Servais L; Department of Paediatrics, MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre, University of Oxford, Oxford, UK.; Department of Paediatrics, Neuromuscular Reference Center, University and University Hospital of Liège, Liège, Belgium.
Jazyk: angličtina
Zdroj: Annals of clinical and translational neurology [Ann Clin Transl Neurol] 2023 Nov; Vol. 10 (11), pp. 2155-2160. Date of Electronic Publication: 2023 Sep 10.
DOI: 10.1002/acn3.51889
Abstrakt: We sought to devise a rational, systematic approach for defining/grouping survival motor neuron-targeted disease-modifying treatment (DMT) scenarios. The proposed classification is primarily based on a two-part differentiation: initial DMT, and persistence/discontinuation of subsequent DMT(s). Treatment categories were identified: monotherapy add-on, transient add-on, combination with onasemnogene abeparvovec, bridging to onasemnogene abeparvovec, and switching to onasemnogene abeparvovec. We validated this approach by applying the classification to the 443 patients currently in the RESTORE registry and explored the demographics of these different groups of patients. This work forms the basis to explore the safety and efficacy profile of the different combinations of DMT in SMA.
(© 2023 The Authors. Annals of Clinical and Translational Neurology published by Wiley Periodicals LLC on behalf of American Neurological Association.)
Databáze: MEDLINE
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