Lupus Overview

Lupus erythematosus is a name given to a collection of autoimmune diseases in which the human immune system becomes hyperactive and attacks healthy tissues.[1] Symptoms of these diseases can affect many different body systems, including joints, skin, kidneys, blood...

Understanding Fragile X

This educational video is produced by Fragile X Association and covers symptoms, diagnosis, and treatments of this rare...

Biomarkers for Type 1 Gaucher Disease | Video

The use of biomarkers can provide valuable insight into the molecular pathogenesis of lysosomal storage diseases such as Gaucher disease and Fabry disease. Dr. Pramod Mistry discusses biomarkers as they pertain to patients with type 1 Gaucher disease. In Gaucher...

Schnitzler syndrome: What you need to know – Mayo Clinic

Discussion of  Schnitzler Syndrome – Video Schnitzler syndrome is a rare disease characterized by a chronic urticarial rash that resists most types of standard therapy. Other symptoms may include fever; bone and joint pain; muscle aches; enlarged lymph nodes,...

Medicines for Rare Diseases

Discussion of Medicines available to treat Rare Diseases – Video The European Medicines Agency plays a central role in the development and authorisation of medicines for rare diseases. These medicines are termed ‘orphan medicines’ in the medical...

Orphan Drugs: Costs

Discussion of Orphan Drug Costs – Video Last week, Nick Bagley argued that the benefits of the Orphan Drug Act are more elusive than commonly assumed. Nick Bagley, a professor of law at the University of Michigan discusses the costs of orphan drugs in this...
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