Individual Treatment Trials—Do Experts Know and Use This Option to Improve the Treatability of Mucopolysaccharidosis?

Mucopolysaccharidoses (MPS) are a group of rare, heterogeneous, lysosomal storage disorders. Patients show a broad spectrum of clinical features with a substantial unmet medical need. Individual treatment trials (ITTs) might be a valid, time- and cost-efficient way to facilitate personalized medicin...

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Detalles Bibliográficos
Autores principales: Wiesinger, Anna-Maria, Strobl, Hannah, Lagler, Florian B.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10058611/
https://www.ncbi.nlm.nih.gov/pubmed/36986515
http://dx.doi.org/10.3390/ph16030416

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10058611/
https://www.ncbi.nlm.nih.gov/pubmed/36986515
http://dx.doi.org/10.3390/ph16030416

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