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

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 therapy m...

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Detalles Bibliográficos
Autores principales: Eskes, Eline C. B., Beishuizen, Cathrien R. L., Corazolla, Eleonore M., van Middelaar, Tessa, Brands, Marion M. M. G., Dekker, Hanka, van de Mheen, Erica, Langeveld, Mirjam, Hollak, Carla E. M., Sjouke, Barbara
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2022
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9587648/
https://www.ncbi.nlm.nih.gov/pubmed/36271424
http://dx.doi.org/10.1186/s13023-022-02543-y

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