PHARMACHON: Pharmacokinetics of VOXZOGO
The following, written by Victoria Garcia, RN, exclusively for The Chandler Project and members of the achondroplasia community are questions and answers compiled from ‘One Family’s Story: A Guide to…
News
Children with achondroplasia to benefit from life-changing treatment added to PBS
The Murdoch Children’s Research Institute has welcomed the news that children born with the most common form of dwarfism, achondroplasia, will have access to a new life-changing treatment on the Pharmaceutical Benefits Scheme (PBS).
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
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
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)…
Tyra Biosciences Expands Development of TYRA-300, an Oral FGFR3 Agent, Into Achondroplasia
In preclinical models, TYRA-300 demonstrated significant increases in growth and bone lengthPhase 2 clinical study for pediatric achondroplasia planned to be initiated in 2024-Conference call and webcast today, March 1st, at 8:00…