by CheckRare Staff | Jan 17, 2023
Gerard Vockley, MD, PhD, Head of the Division of Medical Genetics at UPMC Children’s Hospital of Pittsburgh, discusses new data testing a probiotic to treat individuals with phenylketonuria (PKU). PKU is a rare genetic metabolic disorder that results in...
by CheckRare Staff | Jan 16, 2023
Adam Lamble, MD, Attending Physician, Cancer and Blood Disorders Center, Seattle Children’s Hospital, discusses a recent study that lists six high-risk alterations of acute myeloid leukemia (AML) that ought to be included in Children’s Oncology Group’s...
by CheckRare Staff | Jan 13, 2023
Morie Gertz, MD, Hematologist, Chair Emeritus Internal Medicine, at the Mayo Clinic, gives an overview of immunoglobulin light chain-associated (AL) amyloidosis and how the disease is currently managed. As Dr. Gertz explains, AL amyloidosis is a rare...
by CheckRare Staff | Jan 12, 2023
WHIM syndrome is a rare, congenital primary immunodeficiency disorder associated with neutropenia that typically presents in childhood or adolescence. However, due to the heterogeneous presentation of the disease, coupled with lack of awareness of the condition,...
by CheckRare Staff | Jan 12, 2023
Ryan Jacobs, MD, of the Atrium Health Levine Cancer Institute in Charlotte, NC, provides an overview of the current treatment landscape for patients with chronic lymphocytic leukemia (CLL). CLL is a rare blood cancer resulting in a build-up of lymphocytes in...