QED Achondroplasia Clinical Update
QED Therapeutics, Inc. (“QED”), a subsidiary of BridgeBio Pharma, Inc. (“BridgeBio”) focuses on bringing medicine to people living with conditions that are caused by changes in fibroblast growth factor receptor…
News
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BioMarin Announced Positive Final Results from Vosoritide Phase 3 pivotal trial. (BMN111-301)
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…
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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,…
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Think Beyond the Extremities – Complications in Skeletal Dysplasia
Watch the LLRS 2019 Presidential Guest Lecture: Think Beyond the Extremities – Complications in Skeletal Dysplasia by William G. Mackenzie, MD, FRCSC, FACS.
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Pfizer Acquires Clinical-Stage Biotech Therachon
Expands Pfizer’s rare disease portfolio with potential first-in-class therapy for achondroplasia, a genetic condition and the most common form of short-limb dwarfism NEW YORK & BASEL, Switzerland–Pfizer (NYSE: PFE) today…
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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…
