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BioMarin Receives Positive CHMP Opinion in Europe for Vosoritide for the Treatment of Children with Achondroplasia from Age 2 Until Growth Plates Close
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BioMarin Receives Positive CHMP Opinion in Europe for Vosoritide for the Treatment of Children with Achondroplasia from Age 2 Until Growth Plates Close

European Commission Decision expected Q3 2021Temporary Authorization for Use (ATU) Granted in France to Allow Access and Reimbursement of Vosoritide to Begin ImmediatelyVosoritide is Potentially the First Medicine to be…

Look-Back Study Charts 60 Years of Treatments, Health Characteristics Among People With Dwarfism in Effort to Improve Their Quality Of Life

Look-Back Study Charts 60 Years of Treatments, Health Characteristics Among People With Dwarfism in Effort to Improve Their Quality Of Life

via Johns Hopkins Medicine. In a retrospective study believed to be one of the largest of its kind, researchers say they have successfully charted the health risks, growth patterns, and…

Key Measurement Concepts and Appropriate Clinical Outcome Assessments in Pediatric Achondroplasia Clinical Trials

Key Measurement Concepts and Appropriate Clinical Outcome Assessments in Pediatric Achondroplasia Clinical Trials

Author(s): Natalie Aldhouse, MSc (Presenting Author); Helen Kitchen, MSc (Co-Author); Chloe Johnson, MSc (Co-Author); Chris Marshall, MSc (Co-Author); Hannah Pegram, BSc (Co-Author); Sheryl Pease, MBA PMP (Co-Author); Sam Collins, MD (Co-Author);…