by CheckRare Staff | Mar 6, 2022
As part of the roundtable moderated by Philip Pearl, MD, of Boston Children’s Hospital and Harvard Medical School, the attendees of this virtual discussion focused on key diagnostic aspects of aromatic L-amino acid decarboxylase (AADC) deficiency. Dr. Pearl...
by CheckRare Staff | Mar 3, 2022
Stefan M. Schieke, MD, Assistant Professor of Dermatology at University of Wisconsin-Madison, discusses how cutaneous T-cell lymphomas (CTCL) are typically treated. There are many, multimodal treatment options for the various CTCL subtypes. In addition to...
by CheckRare Staff | Mar 1, 2022
James Howard Jr., MD, Distinguished Professor of Neuromuscular Disease and Professor of Neurology and Medicine at UNC School of Medicine, reviews the treatment landscape for myasthenia gravis. Myasthenia gravis is a chronic autoimmune neuromuscular disease...
by CheckRare Staff | Feb 28, 2022
David Weinstein, MD, Senior Vice President of Clinical Development at Passage Bio, discusses new interim clinical data from the Imagine-1 study of PBGM01, a gene therapy for GM1 gangliosidosis. This data was recently presented at WORLDSymposium 2022. GM1...
by CheckRare Staff | Feb 21, 2022
Stefan M. Schieke, MD, Assistant Professor of Dermatology at University of Wisconsin-Madison discusses cutaneous T-cell lymphoma (CTCL) in special populations such as African Americans and veterans of war. According to a paper published in U.S. Medicine, “The...
