Cystic Fibrosis

Cystic fibrosis (CF) is a genetic disorder that causes mucus to build up and damage organs in the body, particularly the lungs and pancreas. Signs and symptoms may include salty-tasting skin; persistent coughing; frequent lung infections; wheezing or shortness of...

Gene Therapies in Development for Gaucher Disease

  Neil Weinreb, MD, FACP provides a summary of research presented at WORLDSymposium 2020 focused on gene therapy to potentially treat Gaucher disease. Gaucher disease is a rare lysosomal storage disorder in which glucocerebroside accumulates in cells and certain...

COVID-19 and Cancer Treatment

  Eunice Wang, MD, Chief of the Leukemia Service at Roswell Park Comprehensive Cancer Center, talks about the need for cancer patients, like those with acute myeloid leukemia (AML), keep to their regular appointments and treatment schedules during the current...

Diagnosing Angelman Syndrome

  Mathews Adera, MD, Vice President, Neurodevelopment at Ovid Therapeutics discusses some signs and symptoms of Angelman syndrome and the difficulty of diagnosing this rare disorder. Angelman syndrome is a rare genetic disorder affecting roughly 500,000 people...

Still’s Disease (Adult Onset)

Still’s disease is an inflammatory condition characterized by high fevers, rash, sore throat, and joint pain. As it progresses, adult-onset Still’s disease may lead to chronic arthritis and other complications. Still’s disease was named after an...