THE CHANDLER PROJECT JOINS GLOBAL GENES RARE-X PROGRAM
via Murdoch Children’s Research Institute A drug that boosts bone growth in children with achondroplasia may also reduce their chances of sudden infant death syndrome (SIDS), sleep apnoea and needing…
Source: The Lancet Child & Adolescent Health Authors: Prof Ravi Savarirayan, MD β’ Prof William R Wilcox, MD β’ Prof Paul Harmatz, MD β’ John Phillips III, MD β’ Lynda…
The Murdoch Childrenβs Research Institute has welcomed the news that children born with the most common form of dwarfism, achondroplasia, will have access to a new life-changing treatment on the Pharmaceutical Benefits Scheme (PBS).
Did you know that more than 90% ofΒ rare diseasesΒ are still without anΒ FDA-approved treatment? On November 19, 2021, theΒ U.S. Food and Drug Administration approved the first drug to improve growth in…
