Patients’ view on gene therapy development for lysosomal storage disorders: a qualitative study

Patients’ view on gene therapy development for lysosomal storage disorders: a qualitative study

Abstract Introduction Several new treatment modalities are being developed for lysosomal storage disorders (LSDs), including gene therapy. As the currently available treatment options and their influence on disease progression differ greatly within the spectrum of LSDs, willingness to undergo gene t...

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Bibliographic Details
Main Authors: Eline C. B. Eskes, Cathrien R. L. Beishuizen, Eleonore M. Corazolla, Tessa van Middelaar, Marion M. M. G. Brands, Hanka Dekker, Erica van de Mheen, Mirjam Langeveld, Carla E. M. Hollak, Barbara Sjouke
Format: Article
Language:English
Published: BMC 2022-10-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Lysosomal storage diseases
Gaucher disease type 1
Fabry disease
Mucopolysaccharidosis type III
Gene therapy
Qualitative research
Online Access:https://doi.org/10.1186/s13023-022-02543-y

Internet

https://doi.org/10.1186/s13023-022-02543-y

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