by Peter Ciszewski | Aug 12, 2022
Michael L. Wang, MD, Professor, Department of Lymphoma & Myeloma at the University of Texas MD Anderson Cancer Center, discusses results of the phase 3 SHINE study which evaluated the safety and efficacy of ibrutinib in combination with bendamustine and...
by Peter Ciszewski | Aug 10, 2022
Dalia Moawad, MD, Executive Director, Head of Neurological Rare Diseases, Medical Affairs at Genentech, discusses the mechanism of action of risdiplam (Evrysdi), a drug indicated for the treatment of spinal muscular atrophy (SMA) in SMA1 patients 2 months and...
by Peter Ciszewski | Aug 8, 2022
Priya Kishnani, MD, Chief, Division of Medical Genetics and Professor of Pediatrics at the Duke University School of Medicine, discusses the Duke Health Rare Disease Center’s work in Pompe disease. Pompe disease is a rare inherited lysosomal storage disorder...
by Peter Ciszewski | Aug 4, 2022
Mark Forman, MD, PhD, Chief Medical Officer at Passage Bio, discusses the mechanism of action of PBGM01, an investigational gene therapy for infantile GM1 gangliosidosis. GM1 gangliosidosis is an inherited lysosomal storage disorder caused by mutations in the...
by Peter Ciszewski | Jul 27, 2022
Axel Bolte, MSc, MBA, Co-Founder, President, and Chief Executive Officer, Inozyme Pharmaceuticals, gives an overview of ENPP1 deficiency. The ENPP1 gene produces a critical enzyme called ectonucleotide pyrophosphatase/ phosphodiesterase 1 (ENPP1), which...
