This is a Phase 2, multicenter, open-label, dose-escalation and dose-expansion study to evaluate the safety, tolerability, and efficacy of infigratinib, a fibroblast growth factor receptor (FGFR) 1-3-selective tyrosine kinase inhibitor, in children 3 to 11 years of age with Achondroplasia (ACH) who previously participated in the PROPEL study (Protocol QBGJ398-001) for

QED

QED Achondroplasia Clinical Update

FEATURED

QED Therapeutics, Inc. (“QED”), a subsidiary of BridgeBio Pharma, Inc. (“BridgeBio”) focuses on bringing medicine to people living with conditions that are caused by changes in fibroblast growth factor receptor (FGFR) genes. QED is developing a medicine which may improve the overall health and quality of life of people with

FEATURED

BioMarin Announces Positive Final Results from Placebo-Controlled Phase 3 Data in Children with Achondroplasia Treated with Vosoritide Placebo-adjusted Increase in Growth Velocity of 1.6 cm/yr (p<0.0001) in Children Treated with Vosoritide Over One Year Pre-submission Meetings with Health Authorities Planned for H1 2020 to Discuss Marketing Applications SAN RAFAEL, Calif., Dec.

FEATURED

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, Calif., Nov. 14, 2019 /PRNewswire/ — BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) provided an update on its clinical program for vosoritide, an analog of