vosoritide

Vosoritide is a medication used to treat achondroplasia, a genetic disorder characterized by disproportionately short limbs. It is a C-type natriuretic peptide (CNP) analog, meaning it mimics the natural effects of this hormone. Achondroplasia is caused by a mutation in the FGFR3 gene, which leads to overactivity of the receptor and thus inhibited cartilage growth. Vosoritide works by antagonizing this overactive FGFR3 signaling pathway in the growth plates of bones. This helps to promote chondrocyte proliferation and differentiation, ultimately leading to increased linear bone growth. The drug is administered via daily subcutaneous injection. Clinical trials have demonstrated that vosoritide can increase annualized height velocity in children with achondroplasia compared to placebo. It is typically prescribed for children with open growth plates who have not yet reached their final adult height. Vosoritide is a relatively new treatment, and its long-term effects and optimal use are still being studied. It is important for patients to be monitored by healthcare professionals during treatment.