Nusinersen is an antisense oligonucleotide that has been used in the treatment of SMA since 2016. Its method of administration (intrathecal) and the need for repeated injections may be a barrier for some patients.
Researchers studied health data from American patients with SMA, 23 type I, 48 type II and 260 type III, who received their loading dose:
- almost half of them did not stick to the schedule for subsequent injections,
- this poor compliance was correlated with a greater frequency of comorbidities (especially respiratory) and increased consumption of care.
By 24 months, half of the patients had permanently discontinued treatment with nusinersen, mainly due to the high cost of medical care in the United States.
