Improvements in Walking Distance during Nusinersen Treatment - A Prospective 3-year SMArtCARE Registry Study.

ABSTRACT:

Background and objectives

Disease progression in patients with spinal muscular atrophy (SMA) has changed dramatically within the past years due to the approval of three different disease-modifying treatments. Nusinersen was the first drug to be approved for the treatment of SMA patients. Clinical trials provided data from infants with SMA type 1 and children with SMA type 2, but there is still insufficient evidence and only scarcely reported long-term experience for nusinersen treatment in ambulant patients. Here, we report data from the SMArtCARE registry of ambulant patients under nusinersen treatment with a follow-up period of up to 38 months.

Methods

SMArtCARE is a disease-specific registry in Germany, Austria and Switzerland. Data are collected as real-world data during

SUBMITTER: Pechmann A 

PROVIDER: S-EPMC9881023 | biostudies-literature | 2023

REPOSITORIES: biostudies-literature