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New CME Program Highlights Latest Hereditary Angioedema (HAE) Research

by James Radke, PhD | Mar 11, 2022

Jonathan Bernstein, MD, Professor of Medicine at the University of Cincinnati, hosts a 15-minute certified continuing medical education program summarizing the latest information about hereditary angioedema (HAE) that was presented at the American College of Allergy,...

CME Program Highlights the Latest Pyruvate Kinase (PK) Deficiency Research

by James Radke, PhD | Mar 3, 2022

  Rachael Grace, MD, Associate Professor, Harvard Medical School, hosts our latest CME program highlighting the latest research about pyruvate kinase (PK) deficiency presented at ASH 2021 and provides expert analysis of its clinical relevance for busy members of...

Rare Disease Day 2022 – A Day to Embrace Innovation (and Wear Your Stripes)

by James Radke, PhD | Feb 26, 2022

This Monday, many bridges, buildings, water falls, etc will be lit up to celebrate the innovation, courage, fortitude, and inspiration that embodies the rare disease community – from the strength of the patients and caregivers, to the empathy and compassion of health...

Mechanism of Action of Momelotinib to Treat Myelofibrosis 

by James Radke, PhD | Feb 22, 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 mechanism of action of momelotinib, a JAK-inhibitor being investigated as a treatment for...

FDA Approves Mitapivat for Pyruvate Kinase (PK) Deficiency

by James Radke, PhD | Feb 18, 2022

The U.S. Food and Drug Administration (FDA) has approved mitapivat (Pyrukynd) for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency. PK deficiency is a rare blood disorder caused by an inherited mutation in the PKLR gene. It is...
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