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Stakeholders’ views on drug development: the congenital disorders of glycosylation community perspective

BACKGROUND: Congenital disorders of glycosylation (CDG) are a large family of rare genetic diseases for which therapies are virtually nonexistent. However, CDG therapeutic research has been expanding, thanks to the continuous efforts of the CDG medical/scientific and patient communities. Hence, CDG...

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Detalles Bibliográficos
Autores principales:	Monticelli, Maria, Francisco, Rita, Brasil, Sandra, Marques-da-Silva, Dorinda, Rijoff, Tatiana, Pascoal, Carlota, Jaeken, Jaak, Videira, Paula A., dos Reis Ferreira, Vanessa
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2022
Materias:	Research
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9338569/ https://www.ncbi.nlm.nih.gov/pubmed/35907899 http://dx.doi.org/10.1186/s13023-022-02460-0

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