Targeting the Root Cause of Cystic Fibrosis Protein Dysfunction with Dr. Charlotte McKee Sionna TRANSCRIPT
Download PDFFeb 26, 2026
Dr. Charlotte McKee, Chief Medical Officer at Sionna, describes the nature of cystic fibrosis (CF, a genetic disease caused by a mutation in the single CFTR gene. While current CFTR modulator therapies do not address the most common mutation, Sionna's novel oral medicine is designed to target the previously undruggable...
Feb 26, 2026
Dr. Charlotte McKee, Chief Medical Officer at Sionna, describes the nature of cystic fibrosis (CF, a genetic disease caused by a mutation in the single CFTR gene. While current CFTR modulator therapies do not address the most common mutation, Sionna's novel oral medicine is designed to target the previously undruggable...
Effectively Treating Rare Epileptic Disorder Dravet Syndrome with Nayla Chaijale UCB TRANSCRIPT
Download PDFFeb 4, 2026
Nayla Chaijale, Medical Strategy Lead for Rare Syndromes at UCB, describes Dravet syndrome, a rare, medication-resistant epileptic encephalopathy that involves seizures and significant co-morbidities like cognitive and developmental delays. Their approved drug FINTEPLA has a unique mechanism of action that modulates...
Feb 4, 2026
Nayla Chaijale, Medical Strategy Lead for Rare Syndromes at UCB, describes Dravet syndrome, a rare, medication-resistant epileptic encephalopathy that involves seizures and significant co-morbidities like cognitive and developmental delays. Their approved drug FINTEPLA has a unique mechanism of action that modulates...
Feb 2, 2026
Paul Pruitt, Chief Growth Officer at SHARx, which is a company that partners with employers to provide access to drugs not covered by insurance, such as specialty medications. The SHARx model acts as a complementary service to existing Pharmacy Benefit Managers and allows employers to opt out of covering the...