First targeted therapy for children with achondroplasia shows persistent height gain
WASHINGTON--Children with achondroplasia, the most common form of disproportionate short stature, grow taller with trends in improved body proportions after two years of daily vosoritide treatment, a new study analysis finds. Results of the industry-sponsored study will be presented at ENDO 2021, the Endocrine Society's annual meeting.
"This is the first robust evidence of a precision therapy for achondroplasia," said the lead investigator, Ravi Savarirayan, M.D., Ph.D., a professor at Murdoch Children's Research Institute at Royal Children's Hospital in Parkville, Australia.
Achondroplasia is a genetic bone growth ...


