by Madaline Spencer | Apr 13, 2023
Alex Dorenbaum, MD, Chief Medical Officer of Reneo Pharmaceuticals, explains primary mitochondrial myopathies (PMM). As Dr. Dorenbaum explains, PMMs are a group of rare metabolic disorders caused by mutations or deletions in mitochondrial or nuclear DNA. These...
by James Radke, PhD and Madaline Spencer | Apr 13, 2023
Samiah Al-Zaidy, MD, Vice President of Clinical Development and Lead on the Passage Bio GM1 Gangliosidosis (GM1) Program, discusses the updated data from Imagine-1 evaluating PBGM01 in GM1, which was presented at WORLDSymposium 2023. Background of GM1 GM1 is an...
by Madaline Spencer | Apr 11, 2023
Rob Schroeder, a patient with hemophilia B describes his experience receiving gene therapy (etranacogene dezaparvovec). Hemophilia B is a genetic bleeding disorder due to dysfunction or deficiency of coagulation Factor IX. People with this condition may bleed...
by Madaline Spencer | Mar 25, 2023
Julia Warren, MD, Ph.D., Associate Professor of Hematology at the University of Pennsylvania and Hematologist at Children’s Hospital of Philadelphia, gives an overview of chronic neutropenic disorders. Chronic neutropenic disorders are blood disorders...
by Madaline Spencer | Mar 22, 2023
Baseball legend, Bernie Williams, formerly of the New York Yankee (and currently a jazz musician), discusses his advocacy work to raise awareness of interstitial lung diseases, including idiopathic pulmonary fibrosis (IPF). There are IPF is a chronic,...
