by Peter Ciszewski | Feb 13, 2020
Dwight Koeberl, MD, PhD, of Duke University School of Medicine provides an update on the phase 1 study of gene therapy to treat late-onset Pompe disease. As Dr. Koeberl explains in this video, the preliminary results in the low-dose cohort show the gene therapy...
by Peter Ciszewski | Feb 12, 2020
By Fran Sverdrup, PhD, Associate Professor, Department of Biochemistry and Molecular Biology at Saint Louis University. Facioscapulohumeral muscular dystrophy (FSHD) is one of the most common forms of muscular dystrophy. The name itself offers important insights...
by Peter Ciszewski | Feb 12, 2020
Ruth-Anne Langan Pai, PHD Candidate and the collaborative research project lead for the Castleman Disease Collaborative Network (CDCN) provides an overview of Castleman disease. Castleman disease is a group of rare disorders characterized by lymph node...
by Peter Ciszewski | Feb 11, 2020
James Dean, MD, senior medical director at Pharmacyclics provides an update on the most recent long-term data showing ibrutinib to be safe and effective for treating patients with chronic lymphatic leukemia (CLL). CLL is a rare blood cancer resulting in a...
by Peter Ciszewski | Feb 10, 2020
Eric T. Wong, MD, Associate Professor at Harvard Medical School provides an overview of rare cancers. Unlike rare diseases that are defined as affecting less than 200,000 people in the United States, rare cancers are referred to those affecting less than 45,000...
