BioMarin

BioMarin Announces Cumulative Additional Height Gain of 9.0 cm over 54 months versus Natural History in Children with Achondroplasia Treated with Vosoritide in Phase 2 Study

Company Plans to Provide Topline Phase 3 Data by Year End Company Presents Data Confirming that the Phase 3 and Phase 2 Study Participants have Similar Baseline Parameters SAN RAFAEL, Calif., Nov. 14, 2019 /PRNewswire/ — BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) provided an update on its clinical program for vosoritide, an analog of C-type Natriuretic Peptide (CNP), in …

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Ascendis

Ascendis Pharma Announces Orphan Drug Designation Granted for TransCon CNP as Treatment for Achondroplasia

COPENHAGEN, Denmark, Feb. 28, 2019 (GLOBE NEWSWIRE) — Ascendis Pharma A/S (Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative TransCon™ technology to address significant unmet medical needs, today announced that the U.S. Food and Drug Administration(FDA) has granted Orphan Drug Designation (ODD) to TransCon CNP, a long-acting prodrug of C-type natriuretic peptide (CNP) in development for children with achondroplasia. Achondroplasia …

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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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