by CheckRare Staff | Jan 26, 2022
Kathie Bishop, PhD, Chief Scientific Officer at Acadia Pharmaceuticals, discusses the positive top-line results from the pivotal phase 3 Lavender trial testing the efficacy of trofinetide to treat Rett syndrome. Rett syndrome is a rare progressive...
by Peter Ciszewski | Jan 26, 2022
Paula Ragan, PhD, CEO and President, X4 Pharmaceuticals, discusses data presented at The American Society of Hematology Meeting & Exposition (ASH 2021) using artificial intelligence/machine learning model to estimate the prevalence of WHIM syndrome in the...
by Peter Ciszewski | Jan 26, 2022
Stefan M. Schieke, MD, Assistant Professor of Dermatology at University of Wisconsin-Madison, provides an overview of cutaneous T-cell lymphoma (CTCL). Cutaneous T-cell lymphoma belongs to the non-Hodgkin lymphoma class of hematologic T-cell lymphoproliferative...
by Peter Ciszewski | Jan 25, 2022
Paolo Ghia, MD, PhD, Professor at the Università Vita-Salute San Raffaele, Milan, Italy, discusses the updated, long-term data from the phase 2 CAPTIVATE study. This study evaluated ibrutinib plus venetoclax as a first-line treatment for chronic lymphocytic...
by Peter Ciszewski | Jan 24, 2022
David Birch, PhD, Scientific Director at the Retina Foundation of the Southwest, provides an overview of retinitis pigmentosa. As Dr. Birch explains, retinitis pigmentosa is a group of rare, inherited ophthalmological disorders that affect the retina. Retinitis...
