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,…
treatment
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…
Equity for Those Living with a Rare Disease is Equitable Access to Diagnosis, Treatment, Health, Social Care, and Opportunity
Equity for those living with a rare disease is equitable access to diagnosis, treatment, health, social care and opportunity. WHAT IS EQUITY?Equity in practice means meeting people’s specific needs and eliminating barriers preventing their…
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…
Look-Back Study Charts 60 Years of Treatments, Health Characteristics Among People With Dwarfism in Effort to Improve Their Quality Of Life
via Johns Hopkins Medicine. In a retrospective study believed to be one of the largest of its kind, researchers say they have successfully charted the health risks, growth patterns, and…
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,…