Treatment

Children with achondroplasia to benefit from life-changing treatment added to PBS

The Murdoch Children’s 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).

Achondroplasia · BioMarin · Rare Disease Day · Treatment · Voxzogo

More Than 90% of Rare Diseases Are Still Without FDA-Approved Treatment

Did you know that more than 90% of rare diseases are still without an FDA-approved treatment? On November 19, 2021, the U.S. Food and Drug Administration approved the first drug to improve growth in…