Newborn screening for SMA in Southern Belgium

Approval was recently granted for a new treatment for spinal muscular atrophy (SMA). Given that the treatment is effective when administered early and the societal burden of SMA-related disability, the implementation of a newborn screening program is warranted. We describe the stepwise process that...

Disgrifiad llawn

Manylion Llyfryddiaeth
Prif Awduron: Boemer, F, Caberg, J-H, Dideberg, V, Dardenne, D, Bours, V, Hiligsmann, M, Dangouloff, T, Servais, L
Fformat: Journal article
Iaith:English
Cyhoeddwyd: Elsevier 2019