News

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 · TYRA-300

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…

Spinal Stenosis

Fat infiltration in the thigh muscles is associated with symptomatic spinal stenosis and reduced physical functioning in adults with achondroplasia

Svein O. Fredwall, Jennifer Linge, Olga de Vries, Olof Dahlqvist Leinhard, Heidi Beate Eggesbø, Harald Weedon-Fekjær, Mikael Petersson, Per Widholm, Grethe Månum & Ravi Savarirayan Orphanet Journal of Rare Diseases volume 18, Article number: 35 (2023) ABSTRACT BACKGROUNDSymptomatic spinal stenosis is a prevalent complication in…

Achondroplasia · BioMarin · Rare Disease Day · Treatment · Voxzogo

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…

Rare Disease Day

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…