(RTTNews) - BioMarin Pharmaceutical Inc. (BMRN), Wednesday announced positive data from the CANOPY clinical program evaluating Voxzogo in children with achondroplasia, a rare genetic skeletal ...
(RTTNews) - Ascendis Pharma A/S (ASND) Monday said the pivotal ApproaCH study of TransCon CNP, in children with achondroplasia met its primary goal. The company's shares were more than 17 percent up ...
The FDA has granted Breakthrough Therapy Designation for oral infigratinib (BridgeBio Pharma), which is currently under development for children with achondroplasia, according to a press release from ...
BridgeBio said it received breakthrough therapy designation from the U.S. Food and Drug Administration for infigratinib, a treatment under development for children with achondroplasia.
Predictably, weekly vs. daily subcutaneous injections made a big difference in the achondroplasia space, where shares of Copenhagen, Denmark-based Ascendis Pharma A/S (NASDAQ:ASND) closed Sept. 16 at ...