Symptoms and Quality of Life in Patients with Fabry Disease: Results from an International Patient Survey

INTRODUCTION: Fabry disease is an X-linked lysosomal storage disorder caused by a deficiency of α-galactosidase A. Symptoms include neuropathic pain and gastrointestinal problems, such as diarrhoea. To inform and support the design of a Phase III clinical trial for a new oral treatment for Fabry dis...

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Detalles Bibliográficos
Autores principales: Morand, Olivier, Johnson, Jack, Walter, Jerry, Atkinson, Leone, Kline, Gregory, Frey, Aline, Politei, Juan, Schiffmann, Raphael
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer Healthcare 2019
Materias:
Original Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6822826/
https://www.ncbi.nlm.nih.gov/pubmed/31435831
http://dx.doi.org/10.1007/s12325-019-01061-x

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6822826/
https://www.ncbi.nlm.nih.gov/pubmed/31435831
http://dx.doi.org/10.1007/s12325-019-01061-x

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