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BioMarin Announces Positive Final Results from Placebo-Controlled Phase 3 Data in Children with Achondroplasia Treated with Vosoritide Placebo-adjusted Increase in Growth Velocity of 1.6 cm/yr (p<0.0001) in Children Treated with Vosoritide Over One Year Pre-submission Meetings with Health Authorities Planned for H1 2020 to Discuss Marketing Applications SAN RAFAEL, Calif., Dec.

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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

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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

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Achondroplasia

#RareDiseaseDay 2019

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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!