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...
by CheckRare Staff | Feb 17, 2022
Jeffrey A. Zonder, MD, Hematologist-Oncologist from the Barbara Ann Karmanos Cancer Institute in Detroit, Michigan, describes the treatment landscape for patients with relapsed or refractory multiple myeloma. Multiple myeloma is a rare blood cancer associated...
by CheckRare Staff | Feb 15, 2022
David Birch, PhD, Scientific Director at the Retina Foundation of the Southwest, discusses phase 2/3 Sirius and Celeste clinical trials of investigational therapy, QR-421a, for people with USH2A-mediated retinitis pigmentosa and Usher syndrome. Retinitis...
by CheckRare Staff | Feb 9, 2022
James Howard Jr, MD, Distinguished Professor of Neuromuscular Disease and Professor of Neurology and Medicine at UNC School of Medicine, discusses the phase 3 trial of efgartigimod in patients with myasthenia gravis, which recently led to the drug’s approval by...
