Experiences of caregivers of children with spinal muscular atrophy participating in the expanded access program for nusinersen: a longitudinal qualitative study.

ABSTRACT:

Background

Expanded access programs (EAPs) allow patients with serious, life-threatening conditions access to drugs prior to their formal approval. Despite the possible benefits for patients, EAPs present several challenges, including uncertainty regarding a drug's efficacy and safety as well as inequities regarding access to treatment. Although the number of EAPs is growing, the experience of patients participating in EAPs has not yet been studied. In Germany, an EAP for the treatment of Spinal Muscular Atrophy (SMA) with nusinersen ran from December 2016 to May 2017). SMA is a rare, progressive neuromuscular disorder characterized by muscle atrophy and proximal muscle weakness. Insights into patients' and caregivers' experiences could help to improve future EAPs.

Results

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SUBMITTER: Kiefer P 

PROVIDER: S-EPMC7391692 | biostudies-literature | 2020 Jul

REPOSITORIES: biostudies-literature