Amicus Therapeutics is a biopharmaceutical company developing novel, oral therapeutics known as pharmacological chaperones for the treatment of a range of human genetic diseases. The company is currently conducting research in Gaucher, Fabry and Pompe diseases.
The Lysosomal Storage Disease Network is a research consortium of scientists, laboratories, health care professionals and clinics working as networked centers of excellence to improve basic knowledge and understanding of lysosomal disorders.
The National Organization for Rare Disorders (NORD) is a unique federation of voluntary health organizations dedicated to helping people with rare “orphan” diseases and assisting the organizations that serve them. NORD is committed to the identification, treatment, and cure of rare disorders through programs of education, advocacy, research, and service.
National Tay-Sachs & Allied Diseases Association, Inc.
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