Pyrukynd ushers in new era for rare cellular metabolic disease

The approval of the first oral pill to treat a rare anemia heralds the arrival of a new class of agents that could shake up enzyme replacement therapies for metabolic disease.

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Bibliographic Details
Published in:Nature biotechnology 2022-04, Vol.40 (4), p.446-448
Main Author: Harrison, Charlotte
Format: Article
Language:English
Subjects:
Agriculture
Anemia
Bioinformatics
Biomedical and Life Sciences
Biomedical Engineering/Biotechnology
Biomedicine
Biotechnology
Humans
Life Sciences
Metabolic Diseases
Metabolic disorders
News
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Description
Summary:The approval of the first oral pill to treat a rare anemia heralds the arrival of a new class of agents that could shake up enzyme replacement therapies for metabolic disease.
ISSN:1087-0156
1546-1696
DOI:10.1038/s41587-022-01286-2