by Peter Ciszewski | Jun 21, 2022
Axel Bolte, MSc, MBA, Co-Founder, President, and Chief Executive Officer, Inozyme Pharmaceuticals, gives advice to parents of a baby diagnosed with ENPP1 deficiency. The ENPP1 gene produces a critical enzyme called ectonucleotide pyrophosphatase/...
by Peter Ciszewski | Jun 21, 2022
Ann Barbier, MD, PhD, Chief Medical Officer at CAMP4 Therapeutics, discusses positive animal model data from CAMP4’s investigational RNA-based therapy for Dravet syndrome, CMP-SCN (CO-3527). Dravet syndrome is a rare neurological condition that usually appears...
by Peter Ciszewski | Jun 16, 2022
Priya Kishnani, MD, Chief, Division of Medical Genetics and Professor of Pediatrics at the Duke University School of Medicine, discusses the National Organization for Rare Disorders’s (NORD) Rare Disease Centers of Excellence, of which the Duke Health Rare...
by Peter Ciszewski | Jun 13, 2022
Ajay K. Nooka, MD, Associate Professor of Hematology and Medical Oncology at Emory School of Medicine, discusses the updated efficacy and safety results from the phase 1/2 MajesTEC-1 study evaluating teclistamab in patients with relapsed or refractory multiple...
by Peter Ciszewski | Jun 8, 2022
Sumanta K. Pal, MD, Professor in the Department of Medical Oncology & Therapeutics Research and Co-director of the Kidney Cancer Program at the City of Hope Comprehensive Cancer Center, discusses results from Cohorts 3, 4, and 5 of the COSMIC-021 study,...
