Researchers consulted the SMArtCARE registry, which focuses on SMA in Germany, Austria and Switzerland, in the context of innovative therapies available since 2017:
- data from 2,140 SMA patients listed in the registry were analysed,
- 60% of them received treatment with nusinersen, 24% with risdiplam and 11% with gene therapy (onasemnogene aboparvovec),
- two-thirds of treated patients continued with the initial innovative therapy, reflecting their satisfaction (with both its efficacy and mode of administration),
- for the others, switching from one therapy to another most often occurred when a new innovative drug was launched on the market, more for convenience (a more suitable method of administration) than because of a real decrease in the efficacy of their initial treatment.
The authors point out that, according to their data, switching rarely results in any real functional benefit.
