Global Rare Disease Day

February 24, 2009 at 8:00 am Leave a comment


Raptor Pharmaceuticals, San Francisco Bay Area biotech company, will participate in Global Rare Disease Day on February 28, 2009, joining hundreds of patient organizations, government agencies, medical societies and other drug development companies to focus attention on Rare Diseases. A Rare Disease affects fewer than 200,000 Americans, and nearly 7,000 such diseases afflict nearly 30 million Americans, according to the National Institutes of Health (“NIH”).

Global Rare Disease Day website:

Information on U.S. Rare Disease Day on the NORD website


February 28, 2009


Sacramento / San Francisco Bay Area families active in the fight against cystinosis, a rare genetic disorder

Researchers working towards a cure/improved treatment for cystinosis

Nephropathic cystinosis (cystinosis) is a serious lysosomal storage disease, primarily diagnosed in early childhood. If left untreated, cystinosis destroys major organ systems including the kidneys, eyes, liver, muscles, pancreas and the brain. The current treatment, cysteamine therapy, may be effective at preventing kidney failure by delaying kidney transplants in cystinosis patients. However, patient compliance is challenging due to frequent dosing and potential gastrointestinal side effects.

Raptor Pharmaceuticals’ DR Cysteamine product candidate is a delayed-release oral formulation of cysteamine bitartrate currently being investigated for its potential to improve the management of cystinosis by potentially reducing frequency of dosing and minimizing gastrointestinal side effects.

About Raptor Pharmaceuticals Corp.

Raptor Pharmaceuticals Corp. (“Raptor”) is dedicated to speeding the delivery of new treatment options to patients by working to improve existing therapeutics through the application of highly specialized drug targeting platforms and formulation expertise. Raptor focuses on underserved patient populations where it can have the greatest potential impact. Raptor currently has product candidates in clinical development to treat nephropathic cystinosis, non-alcoholic steatohepatitis (“NASH”), Huntington’s Disease (“HD”) which is also a rare disease, and aldehyde dehydrogenase (“ALDH2”) deficiency.

Raptor’s preclinical programs are based upon bioengineered novel drug candidates and drug-targeting platforms derived from the human receptor-associated protein (“RAP”) and related proteins that are designed to target cancer, neurodegenerative disorders and infectious diseases.

Raptor Pharmaceuticals Corp



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