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…
BioMarin
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…
More Than 90% of Rare Diseases Are Still Without FDA-Approved Treatment
Did you know that more than 90% of rare diseases are still without an FDA-approved treatment? On November 19, 2021, the U.S. Food and Drug Administration approved the first drug to improve growth in…
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…