The Supplemental New Drug Application is Based on Positive Results from Global Randomized Phase 2 Study in Infants…
– In the highest dose level (Cohort 5, 0.25 mg/kg once daily), the mean change from baseline in…
March 2, 2023
Tyra Biosciences Expands Development of TYRA-300, an Oral FGFR3 Selective Agent, into Achondroplasia
– In preclinical models, TYRA-300 demonstrated significant increases in growth and bone length--Phase 2 clinical study for pediatric…
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…
Did you know that more than 90% of rare diseases are still without an FDA-approved treatment? On November 19, 2021, the U.S.…
PAID ACHONDROPLASIA NON-TREATMENT RESEARCH STUDY TO EVALUATE PEN-BASED INJECTION DEVICE When living with a rare disease or condition, there are many opportunities…
Established in 2018, The Chandler Project provides advocacy, awareness, research and support to those affected by achondroplasia, the most common form of dwarfism, and other skeletal dysplasias. TCP hosts the only event in North Amercia that brings together members of the skeletal dysplasia community physicians, researchers, biotech industries and more together at the annual Achondroplasia + Skeletal Dysplasia Research Conference.