Important Considerations When Treating AML

  Andrew Krivoshik, MD, PhD, Oncology Therapeutic Area Head at Astellas Pharma gives an overview of treatment options for patients with acute myeloid leukemia (AML).  AML is an aggressive rare cancer of the blood and bone marrow with a 5-year survival rate of...

Advice From a Niemann-Pick Disease Mom: Avoid Google

  Taylor Sabky, mother of a 3-year-old boy who had Niemann-Pick disease type A, explains how she came to learn about this devastating disease – beginning with advice from her doctor to not google it while they confirmed a diagnosis. Taylor worked tirelessly to...

What is AADC Deficiency?

  Irina Anselm, MD, from Boston Children’s Hospital provides an overview of aromatic L-amino acid decarboxylase (AADC) deficiency. AADC deficiency is a rare, genetic disorder caused by defect in the dopa decarboxylase (DDC) gene that leads to a reduction in the...

Clinical Trials for Lupus Nephritis

  Richard A. Furie, MD, from Northwell Health explains the planning and execution of clinical trials for lupus nephritis. Lupus nephritis is an inflammation of the kidneys caused by systemic lupus erythematosus (SLE). As Dr. Furie explains in this video, clinical...

Favorite Behind the Mystery Episodes

  Molly Mager and Carri Levy, co-creators of Behind the Mystery, talk about their favorite episodes. Behind the Mystery is a recurring segment on Lifetime Channel’s The Balancing Act that is devoted to rare and genetic conditions. The two producers have created...