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

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

Abstract 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. Hen...

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Bibliographic Details
Main Authors: Maria Monticelli, Rita Francisco, Sandra Brasil, Dorinda Marques-da-Silva, Tatiana Rijoff, Carlota Pascoal, Jaak Jaeken, Paula A. Videira, Vanessa dos Reis Ferreira
Format: Article
Language:English
Published: BMC 2022-07-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Drug development
People-centricity
Congenital disorder(s) of glycosylation (CDG)
Electronic (e-)survey
Patient-reported outcome measures
Online Access:https://doi.org/10.1186/s13023-022-02460-0

Internet

https://doi.org/10.1186/s13023-022-02460-0

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