Chinese researchers have gone through the data published by the American FDA with great care concerning the clinical and biological safety of three drugs commonly used in the treatment of proximal spinal muscular atrophy linked to the SMN1 gene (SMA):
- these researchers used a Venn-type comparative method based on data from the FDA’s pharmacovigilance system,
- their study included 5,324 SMA patients treated with nusinersen, 1,184 treated with risdiplam and 1,277 treated with gene therapy,
- 27 types of side effect were observed in all three cases,
- 196 were more specific to nusinersen but included adverse reactions during lumbar punctures,
- 143 side-effects were more significantly present with gene therapy, including increased transaminases and variations in troponin or lactic acid.
These very useful studies highlight the specificities of the respective profiles of good and poor tolerance of the three medications.
