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A recent publication in Genes & Diseases has delivered a compelling synthesis of the latest insights into the cellular ...
A recent publication in Genes & Diseases has delivered a compelling synthesis of the latest insights into the cellular ...
A landmark review in Nature Reviews Genetics dissects the rare and common genetic variants that shape human height. The paper ...
Achondroplasia is a rare genetic disorder of bone growth that causes short-limbed dwarfism. It occurs due to mutations in a single gene called the FGFR3.
The oral FGFR1-3 selective tyrosine kinase inhibitor infigratinib was found to be safe and effective at the highest studied dose among children with achondroplasia in a phase II dose-finding study.
TransCon CNP is an investigational prodrug of C-type natriuretic peptide (CNP) administered once weekly and designed to treat individuals with achondroplasia by providing continuous exposure of ...