8 questions for a thorough history to diagnose migraines

Submission is based on 3 clinical trials, including data from the ApproaCH Trial among children with achondroplasia.

Jamie Glater, MD, discusses an NIH-sponsored study investigating the effectiveness of an auditory brain stem implant in young children with congenital cochlear nerve agenesis.

The PANDAS/PANS questionnaire is based on symptoms and comorbidities described in the literature and is a self-report completed by parents of children younger than 18.

Results were from the phase 3 STEER study among a broad population of patients with SMA aged 2 to under 18 years.

First approved for PNH in 2007, the monoclonal antibody is now the first and only treatment for pediatric patients living with gMG.

The FDA has accepted PTC Therapeutics' NDA for vatiquinone, a potential treatment for Friedreich's ataxia, with a decision expected by August 19, 2025.