Fetal therapies and trials for lysosomal storage diseases: a survey of attitudes of parents and patients

BACKGROUND: Lysosomal storage diseases (LSDs) are inherited metabolic disorders that may lead to severe multi-organ disease. Current ERTs are limited by anti-drug antibodies, the blood–brain barrier, and early disease onset and progression before ERT is started. We have opened a phase I clinical tri...

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Detalles Bibliográficos
Autores principales: Schwab, Marisa E., Brown, Julia E. H., Lianoglou, Billie, Jin, Chengshi, Conroy, Patricia C., Gallagher, Renata C., Harmatz, Paul, MacKenzie, Tippi C.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2022
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8800365/
https://www.ncbi.nlm.nih.gov/pubmed/35093147
http://dx.doi.org/10.1186/s13023-022-02178-z

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