PEDIATRIC PUZZLER A 17-month-old with stiffening episodes: No red flags seen flying

ConSynance Therapeutics' CSTI-500, an innovative Triple Monoamine Reuptake Inhibitor, has received FDA's Rare Pediatric Disease Designation for treating Prader-Willi Syndrome in children.

Initial approval for maralixibat was granted on March 13, 2024, to treat PFIC patients aged 5 years and older.

The third and final episode in our series looks at what is in the pipeline as well as a discussion around FDA guidance.

Merck announced clesrovimab met all primary safety and efficacy endpoints, with additional detailed findings to be presented at an upcoming scientific congress.