News

New Drug Treats Dwarfism
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New Drug Treats Dwarfism

Life-changing breakthrough for children Murdoch Children’s Research Institute (MCRI) recently shared a new article from the Herald Sun titled ‘New Drug Treats Dwarfism’ written by Sue Dunlevy. In the post,…

FDA Accepts BioMarin’s Supplemental New Drug Application to Expand Use of VOXZOGO® (vosoritide) for Injection to Treat Children with Achondroplasia Under the Age of 5
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FDA Accepts BioMarin’s Supplemental New Drug Application to Expand Use of VOXZOGO® (vosoritide) for Injection to Treat Children with Achondroplasia Under the Age of 5

The Supplemental New Drug Application is Based on Positive Results from Global Randomized Phase 2 Study in Infants and Young Children FDA set PDUFA Target Action Date of October 21, 2023…

BridgeBio Announces Positive Phase 2 Cohort 5 Results of Infigratinib in Achondroplasia Demonstrating Mean Increase in Annualized Height Velocity of 3.03 cm/yr with No Treatment-Related Adverse Events
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BridgeBio Announces Positive Phase 2 Cohort 5 Results of Infigratinib in Achondroplasia Demonstrating Mean Increase in Annualized Height Velocity of 3.03 cm/yr with No Treatment-Related Adverse Events

In the highest dose level (Cohort 5, 0.25 mg/kg once daily), the mean change from baseline in annualized height velocity (AHV) at six months was +3.03 cm/yr (p = 0.0022)…