PKU Treatment: Medical Foods vs Kuvan vs Palynziq

  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...

How Is AL Amyloidosis Currently Treated?

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...

Recognizing and Diagnosing WHIM Syndrome

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,...