by CheckRare Staff | Aug 21, 2023
Bob Lojewski, CSL Behring’s senior vice president and general manager of North America, talks about the changes in the treatment landscape for hemophilia B, including the recent use of gene therapy. Hemophilia B is a rare bleeding disorder caused by...
by CheckRare Staff | Aug 17, 2023
Patrick Lewis, PharmD, Senior Director, Medical Affairs at Amryt Pharma discusses the treatment options for patients with acromegaly. Acromegaly is a rare endocrine disorder due to excess levels of growth hormone (GH). In many cases, a benign...
by CheckRare Staff | Aug 15, 2023
Cary Harding, MD, at Oregon Health & Science University (OHSU), provides an overview of phenylketonuria (PKU). Toxic levels of the amino acid phenylalanine (Phe) cause PKU, a rare metabolic disease that can result in neurocognitive deficits....
by CheckRare Staff | Aug 14, 2023
Patrick Lewis, PharmD, Senior Director, Medical Affairs at Amryt Pharma provides an overview of acromegaly and why diagnostic delays for this rare disease are so common. Acromegaly is a rare endocrine disorder caused by excess levels of growth hormone...
by CheckRare Staff | Aug 11, 2023
Andrea Meredith, Ph.D., Professor of Physiology at the University of Maryland School of Medicine, explains KCNMA1-linked channelopathy. KCNMA1-linked channelopathy is a rare neurological disorder due to mutations in the KCNMA1 gene. That gene encodes the...
