Pfizer Cuts Recifercept for Achondroplasia Program
Pfizer punts pair of orphan drugs amid rare disease R&D rethink Pfizer, amid a rethink of its rare disease R&D strategy, has punted (PDF) a pair of orphan drug candidates from its…
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European Medicines Agency Validates Application for Extension of Indication for VOXZOGO® (vosoritide) for injection to Treat Children with Achondroplasia Under the Age of 2
BioMarin Submits Supplemental New Drug Application to U.S. Food and Drug Administration to Expand Label to Treat Children with Achondroplasia Under the Age of 5 Submissions Based on Favorable Results…
BridgeBio Pharma Announces Positive Interim Results from a Phase 2 Trial of Infigratinib in Achondroplasia Demonstrating an Increase in Annualized Height Velocity of 1.52 cm/year in Children 5 Years of Age and Older, and Adds 5th Cohort to Trial
PRESS RELEASE OBTAINED FROM BRIDGEBIO SITE – INVESTOR.BRIDGEBIO.COM – At the highest dose level evaluated to date (Cohort 4, 0.128 mg/kg once daily), the mean change from baseline in annualized…
4th Annual Achondroplasia Research Conference
Last week, The Chandler Project (TCP) hosted the 4th Annual Achondroplasia Research Conference in Chicago, IL. Although this was the fourth conference, it was only the third one where we…
Clinical Trials Day 2022
Did you know May 20 is Clinical Trials Day? Clinical Trials Day is a day to dedicated to raising awareness of clinical trials and also to say ‘thank you’ to…
Achondroplasia · Ascendis · BioMarin · Hypochondroplasia · Limb Lengthening · Pfizer · QED · Skeletal Dysplasia
The Perfect Date: National DNA Day
The perfect date: April 25th…because it’s not too hot, not too cold. All you need is a light jacket…Oh! And it’s also #NationalDNADay! National DNA Day is a global movement…