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First targeted therapy for children with achondroplasia shows persistent height gain – Medical Xpress

Children with achondroplasia, the most common form of disproportionate short stature, grow taller with trends in improved body proportions after two years of daily…

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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…

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