Children with achondroplasia who are treated for the condition currently face daily or weekly injections, a demand that falls on families for years. A phase 3 trial of an oral alternative has now drawn an unusually direct endorsement from a specialist writing in the New England Journal of Medicine.
Dr. Isidro B. Salusky of UCLA Health described the trial results as "a potentially giant step forward" in an editorial published in the journal on September 2. The full commentary appears among the journal's featured content. Salusky is a distinguished professor of pediatrics at the David Geffen School of Medicine at UCLA and chief of pediatric nephrology. The editorial was co-written with Dr. Harald Jueppner of Massachusetts General Hospital.
