The Belgian pilot programme for newborn screening in SMA is called Sun May Arise on SMA. Sun May Arise on SMA offers a 3-year neonatal screening programme in Wallonia, Belgium, and is currently in its final year.
The Belgian pilot was launched by Prof. Laurent Servais and is managed by Tamara Dangouloff. They already have one publication available about their work called Newborn Screening for SMA in Southern Belgium with future publications planned. You can follow them on Facebook here.
"The objective of this research is to demonstrate the feasibility of neonatal screening for spinal muscular atrophy and its medical and financial impact in a European state.
The various clinical developments in progress in spinal muscular atrophy converge to demonstrate the importance of the early nature of the treatment implemented. Identifying pre-symptomatic patients is therefore crucial, not only to improve the effectiveness of treatment on an individual level, but also to reduce the societal cost of the disability of patients treated post-symptomatically."
Translated into English from the Sun May Arise Facebook Page
The Belgian Pilot project was conducted as a three year study, limited to Southern Belgium, as governance infrastructure for SMA newborn screening programme are different for the two communities in the country (Federation Wallonie Bruxelles and Vlaams Governement).
This pilot included three of the eight neuromuscular reference centres in Belgium. Awareness and political support for NBS in SMA was sought through meetings and media coverage and presented to the boards of the governmental agency in charge of NBS in Southern Belgium (Office de la Naissance et de l’Enfance (ONE), which issued a positive opinion on the project an included SMA in the list of disorders to be evaluated during the future revision of the NBS core panel in Southern Belgium. The whole process was strongly supported by the Belgian patient advocacy group Association Belge contre les Maladies Musculaires or ABMM.
The project was initially funded through a private donation. Subsequent support was provided by the patient organisation ABMM and other private donations addressed to ABMM and directed to the project. Additionally, grants were awarded from the Southern Belgium Ministry of Childhood and Investigator Initiated Trials were conducted by AveXis (a Novartis company), Biogen, and Roche.
General information on the NBS is systematically provided to all pregnant women, according to Southern Belgium current local legislation, referring to the website of the public agency for the list of screened diseases. While NBS is strongly recommended, it is not mandatory in Southern Belgium: Parents have the right to refuse the newborn screening for their child. Such opting-out, which happens extremely rarely, is not disease-specific and concern the entire programme (Boemer F et al, Neuromuscular Diosrders 2019).
The Belgian pilot programme will finish on 1 March 2021, and the newborn screening activity for SMA will hopefully continue in Wallonia under the public health system when a new law is passed. They have already detected and treated children pre-symptomatically, with some remarkable results.
The SMA NBS Alliance would like to thank Prof. Laurent Servais, Dr. Nathalie Goemans and Tamara Dangouloff for their continued commitment to families affected by SMA.