Achondroplasia

The Chandler Project

FEATURED

As our first project, we are currently working on a weekend event dedicated to learning about new research and surgical options for achondroplasia. Our goal is to then turn this into a bigger event where new research and medical advancements are presented for other forms of skeletal dysplasias, including achondroplasia, as

FEATURED

SAN RAFAEL, Calif., June 14, 2018 /PRNewswire/ — BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced today that the company dosed the first participant in a global Phase 2 study for vosoritide, an analog of C-type Natriuretic Peptide (CNP), in infants and young children with achondroplasia, the most common form of disproportionate short stature in humans.

FEATURED

BASEL, Switzerland, February 14, 2018 – Therachon AG, a biotechnology company focused on rare genetic diseases, today announced the dosing of the first subject in its Phase 1 trial for TA-46, a soluble recombinant human fibroblast growth factor receptor 3 (FGFR3) ligand trap. TA-46 works by normalizing the overactive FGFR3 signaling