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

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 we...

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Bibliographic Details
Main Authors: Petra Kiefer, Janbernd Kirschner, Astrid Pechmann, Thorsten Langer
Format: Article
Language:English
Published: BMC 2020-07-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Spinal muscular atrophy
Nusinersen
Expanded Access program
Compassionate care program
Qualitative research
Interview study
Online Access:http://link.springer.com/article/10.1186/s13023-020-01477-7

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http://link.springer.com/article/10.1186/s13023-020-01477-7

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