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...
by CheckRare Staff | Feb 3, 2022
Philippe Duchateau, PhD, Chief Scientific Officer at Cellectis, describes the company’s TALEN gene editing technology and its application in hematologic cancers. TALEN is based on a class of proteins derived from transcription activator-like effectors (TALEs),...
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...
