by CheckRare Staff | Aug 11, 2022
Barbara Burton, MD, Attending Physician, Genetics, Birth Defects & Metabolism, Ann & Robert H. Lurie Children’s Hospital of Chicago, discusses the diagnostic challenges often faced by alpha mannosidosis patients. Alpha mannosidosis is a multisystemic,...
by CheckRare Staff | Aug 9, 2022
Srdan Verstovsek, MD, PhD, Medical Oncologist and Professor in the Department of Leukemia at The University of Texas MD Anderson Cancer Center, discusses the need for clinicians to be more optimistic about the outcomes of patients with myelofibrosis. ...
by CheckRare Staff | Aug 1, 2022
Susan L Samson, MD, PhD, FRCPC, FACE, Chair of Endocrinology at Mayo Clinic in Jacksonville, Florida, discusses positive efficacy data from the open-label extension of the OPTIMAL study of oral octreotide, an orphan drug that was approved for the treatment of...
by CheckRare Staff | Jul 28, 2022
Jennifer McNary, Executive Director, Head of Patient Advocacy and Engagement at Fulcrum Therapeutics, and mother to two Duchenne muscular dystrophy patients, discusses the significance of using reachable workspace (RWS) as the primary outcome measure for REACH,...
by CheckRare Staff | Jul 12, 2022
Ricardo Dolmetsch, PhD, President of Research and Development at uniQure, discusses positive data from the low-dose cohort of the ongoing phase 1/2 clinical trial of AMT-130, the first-ever AAV gene therapy for Huntington’s disease. Huntington disease is an...
