FDA Approves Otezla for Oral Ulcers Associated With Behçet’s disease

The FDA approved apremilast for the treatment of adult patients with oral ulcers associated with Behçet’s disease, a rare chronic multisystem inflammatory disease. The FDA based its approval on the RELIEF study, a phase 3 randomized, double-blind, placebo-controlled...

Lynn O’Connor Vos Discusses the Muscular Dystrophy Association

Lynn O’Connor Vos, President and Chief Executive Officer at the Muscular Dystrophy Association, discusses her organization and it’s approach to funding research to accelerate treatments and cures across the full spectrum of neuromuscular diseases.

Typical Physical Features Across All SubTypes of Mucopolysaccharidoses (MPSs)

Heather A. Lau, MD, Director, Lysosomal Storage Disease Program at NYU Langone Health, discusses some of the typical physician features across all subtypes of ,mucopolysaccharidoses (MPSs), a group of rare, inherited lysosomal storage disorders that are clinically characterized by abnormalities in multiple organ systems and reduced life expectancy.

Peter Saltonstall Discusses the Orphan Drug Act

Peter Saltonstall, President and Chief Executive Officer of the National Organization for Rare Disorders (NORD), discusses his organization’s position on the Orphan Drug Act.

2030 Goal: Universal Health Coverage for Rare Diseases

Rare Disease International (RDI) is a global alliance of rare disease patient organizations designed to be an international voice for the rare disease community. One of its key initiatives is to include rare diseases in the Universal Health Coverage being developed by the United Nations (UN) for the year 2030.

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