C-type natriuretic peptide–based therapy explicitly addresses the underlying pathophysiologic mechanisms of achondroplasia by down-regulating FGFR3 signaling through the MAPK pathway and ...

In this phase 2 dose-finding study, we evaluated the safety and efficacy of oral infigratinib in children with achondroplasia between the ages of 3 and 11 years. A total of 72 children were ...

“The receipt of Breakthrough Therapy Designation, the first ever for a treatment being developed for children with achondroplasia, marks another important milestone for our skeletal dysplasia ...

The mean change from baseline at 18 months in height z score was 0.54 (95% CI 0.35-0.72) relative to an untreated achondroplasia reference population, while the mean change from baseline in upper ...

More information: Ravi Savarirayan et al, Oral Infigratinib Therapy in Children with Achondroplasia, New England Journal of Medicine (2024). DOI: 10.1056/NEJMoa2411790 ...

A phase 3 study of the investigational drug vosoritide establishes it as the first precision treatment for children with achondroplasia, the most common type of dwarfism, say researchers.

A phase three global clinical trial has shown that the drug vosoritide restores close-to-average bone growth rates of children with achondroplasia, the most common form of dwarfism. The study ...

The FDA has expanded its accelerated approval of an injectable therapy for treating achondroplasia to include children of all ages, according to a press release. The FDA approved a new indication ...

A promising daily tablet is effective at increasing height and improving proportional limb growth in children with achondroplasia, the most common form of dwarfism, according to a new study.

RIBOMIC Announces Completion of Patient Enrollment in Early Phase II Study of umedaptanib pegol for Achondroplasia and Initiation of Dosing in Cohort 2 of Early Phase II Clinical Study ...

RIBOMIC Announces Positive Interim Results from Phase IIa Trial of umedaptanib pegol in Achondroplasia Demonstrating Increase in Annualized Growth Rate of up to +4.6 cm/year in Children 5 Years of ...