Dr ravi savarirayan biography of barack
•
BioMarin to Present Data Underscoring Sustained positiv Impact of VOXZOGO® (vosoritide) on Health-Related Quality of Life, Growth and Maintenance of Bone Strength in Children with Achondroplasia at 2024 International Skeletal Dysplasia Society Meeting
Results from Ongoing CANOPY Clinical Program for VOXZOGO Showcase Impact of C-Type Natriuretic Peptide (CNP) as mästare Regulator of Growth-Related Development Across Multiple Genetic Skeletal Conditions
New uppgifter Highlight Meaningful Improvements in Health-Related Quality of Life for Children with Achondroplasia
Investigator-Led Study Shows Significant Increases in Bone Length While Maintaining Bone Strength in Children with Achondroplasia
Investigator-Led Study Observes Sustained Growth Gains in Children with Hypochondroplasia, Noonan Syndrome and Genetic Variants Associated with Idiopathic Short Stature
SAN RAFAEL, Calif., Sept. 18, 2024 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that p
•
Moeen AlSayed is a Consultant Clinical Geneticist, working in Clinical Biochemical Genetics and Metabolic Diseases at King Faisal Specialist Hospital and Research Centre in Riyadh, Kingdom of Saudi Arabia and a Professor of Genetics at Al-Faisal University in Riyadh. Professor AlSayed has established and is the Director of the MSc Genetic Counselling Programme at AlFaisal University.
Professor AlSayed is a previous Chair of the Department of Medical Genetics at King Faisal Specialist Hospital and Research Centre in Riyadh. He served in this position for 11 years. During his tenure as a Chair and among many achievements, he has established a completely new and unique Adult Genetics and Metabolic Diseases service and a fully dedicated genetic counselling service in the entire Gulf region. The majority of the practicing genetic counsellors in Saudi Arabia are graduates of the MSc Genetic Counselling Programme that was established by Professor AlSayed in 2014.
Professor AlSayed obta
•
BridgeBio Announces First Child Dosed in PROPEL 3, its Phase 3 Clinical Trial for Infigratinib in Children with Achondroplasia
– BridgeBio has dosed the first child in PROPEL 3, a one-year, 2:1 randomized, placebo-controlled Phase 3 pivotal trial evaluating the efficacy and safety of infigratinib in children with achondroplasia
– The U.S. Food and Drug Administration (FDA) and European Union (EU) European Medicines Agency (EMA) shared positive feedback that the PROPEL 3 trial design would be acceptable as a registrational study to support a marketing application
– Data from PROPEL 2, BridgeBio’s Phase 2 clinical trial of infigratinib in children with achondroplasia, supports infigratinib’s potential as an oral, well-tolerated treatment option; results shared at ENDO 2023 included a significant increase in annual height velocity (AHV) with a mean change of +3.38 cm/year from baseline at six months, and early but promising trends towards improvement in propo