PHARMACHON • JUNE 16-18 • CHICAGO, IL
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).
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,…
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…
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)…
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…
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…