Kirschner, J;
Butoianu, N;
Goemans, N;
Haberlova, J;
Kostera-Pruszczyk, A;
Mercuri, E;
van der Pol, WL;
... Muntoni, F; + view all
(2020)
European ad-hoc consensus statement on gene replacement therapy for spinal muscular atrophy.
European Journal of Paediatric Neurology
10.1016/j.ejpn.2020.07.001.
(In press).
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Abstract
Spinal muscular atrophy (SMA) used to be one of the most common genetic causes of infant mortality. New disease modifying treatments have changed the disease trajectories and most impressive results are seen if treatment is initiated in the presymptomatic phase of the disease. Very recently, the European Medicine Agency approved Onasemnogene abeparvovec (Zolgensma®) for the treatment of patients with SMA with up to three copies of the SMN2 gene or the clinical presentation of SMA type 1. While this broad indication provides new opportunities, it also triggers discussions on the appropriate selection of patients in the context of limited available evidence. To aid the rational use of Onasemnogene abeparvovec for the treatment of SMA, a group of European neuromuscular experts presents in this paper eleven consensus statements covering qualification, patient selection, safety considerations and long-term monitoring.
| Type: | Article |
|---|---|
| Title: | European ad-hoc consensus statement on gene replacement therapy for spinal muscular atrophy |
| Location: | England |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.1016/j.ejpn.2020.07.001 |
| Publisher version: | http://dx.doi.org/10.1016/j.ejpn.2020.07.001 |
| Language: | English |
| Additional information: | This version is the version of record. For information on re-use, please refer to the publisher’s terms and conditions. |
| Keywords: | Gene therapy, Nusinersen, Onasemnogene abeparvovec, SMN1, SMN2, Spinal muscular atrophy, Zolgensma |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Developmental Neurosciences Dept |
| URI: | https://discovery.ucl.ac.uk/id/eprint/10110722 |
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