NEW PODCAST: The CheckRare Brief + +

NEW PODCAST: The CheckRare Brief Episode 2: FDA Approves New Narcolepsy Treatment On this week’s episode of The CheckRare Brief, we discuss FDA’s approval of Orzeyful (oveporexton) to treat patients with narcolepsy type 1, CAMP4’s first-in-human clinical trial for patients with SYNGAP1-related disorders, and BioMarin’s termination for their ENPP1 deficiency program following mixed results from their phase 3 clinical trial. https://checkrare.com/fda-approves-new-narcolepsy-treatment-the-checkrare-brief/ Episode 1: FDA Advisory Board’s Concerns About New Duchenne Drug, Brain Fog in Immune Thrombocytopenia https://checkrare.com/fda-advisory-boards-concerns-about-new-duchenne-drug-brain-fog-in-immune-thrombocytopenia/ The CheckRare Brief delivers a concise summary of the week’s most important developments in rare diseases. Each episode covers FDA approvals, clinical trial updates, conference highlights, scientific publications, and industry news that matter to healthcare professionals, researchers, advocates, and the rare disease community.