PRESENTATION · Dec 2021 · PDF

BBIO--CAH-GTx-BBP-631--ASGCT-2021--NHP

PRESENTATION · Dec 2021 · PDF

BBIO--CAH-GTx-BBP-631--ENDO-2021--Phase-1-2-Study-Design

PUBLICATION · May 2021 · PDF

Genetics in Medicine, Achondroplasia Natural History Study (CLARITY): A Multicenter Retrospective Cohort Study of Achondroplasia in the United States

PUBLICATION · Feb 2021 · PDF

Bone, Lifetime Impact of Achondroplasia: Current Evidence and Perspectives on the Natural History

PRESENTATION · Dec 2019 · PDF

BBIO--CAH-GTx-BBP-631--ESGCT-2019--NHP

PUBLICATION · Jan 2019 · PDF

Orphanet Journal of Rare Diseases, Achondroplasia: A Comprehensive Clinical Review

PUBLICATION · Aug 2018 · PDF

Nature Communications, Ribitol Restores Functionally Glycosylated Alpha-dystroglycan and Improves Muscle Function in Dystrophic FKRP-mutant Mice

PUBLICATION · Dec 2016 · PDF

Developmental Dynamics, Achondroplasia: Development, Pathogenesis, and Therapy

PUBLICATION · May 2016

The Journal of Clinical Investigation, Tyrosine Kinase Inhibitor NVP-BGJ398 Functionally Improves FGFR3-related Dwarfism in Mouse Model