Understanding and modifying Fabry disease: Rationale and design of a pivotal Phase 3 study and results from a patient-reported outcome validation study

The use of available treatments for Fabry disease (FD) (including enzyme replacement therapy [ERT]) may be restricted by their limited symptom improvement and mode of administration. Lucerastat is currently being investigated in the MODIFY study as oral substrate reduction therapy for the treatment...

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Detalles Bibliográficos
Autores principales: Wanner, Christoph, Kimonis, Virginia, Politei, Juan, Warnock, David G., Üçeyler, Nurcan, Frey, Aline, Cornelisse, Peter, Hughes, Derralyn
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2022
Materias:
Research Paper
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9248229/
https://www.ncbi.nlm.nih.gov/pubmed/35782623
http://dx.doi.org/10.1016/j.ymgmr.2022.100862

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9248229/
https://www.ncbi.nlm.nih.gov/pubmed/35782623
http://dx.doi.org/10.1016/j.ymgmr.2022.100862

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