Achondroplasia

#RareDiseaseDay 2019

Out now – new official #RareDiseaseDay video! Watch Filip and Alina’s story. He is from Romania and lives with achondroplasia Share the video to raise awareness of rare diseases for Rare Disease Day 2019. #ShowYourRare Rare disease day is on February 28!

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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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