Achondroplasia

The Chandler Project

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 well as (or and) congenital …

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BioMarin

BioMarin Doses First Participant in Phase 2 Study of Vosoritide for Treatment of Infants and Young Children with Achondroplasia

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. “Vosoritide represents an innovative therapy …

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Therachon

Therachon Announces Dosing of First Subject in Phase 1 Clinical Trial Evaluating TA-46, a Novel Investigational Therapy for the Potential Treatment of Achondroplasia

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 pathways that underlie bone abnormalities …

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