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Pompe |
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RESEARCH |
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Pompe disease is caused by a defective or missing enzyme that breaks down
glycogen. If glycogen builds up. the muscles degenerate & contribute to heart failure. Genzyme, Cambridge, MA, USA has begun trials allowed under the 1983 Orphan Drug Act allowing drugs for rare diseases per 8/26/03 Wall Street Journal www.wsj.com |
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The Cure - How a Father Raised $100 Million - and Bucked the Medical
Establishment - In a Quest to Save His Children
Anand,
Geeta by Regan publishers 8/30/2006 www.wsj.com editorial about Patrick Crowley (and family with 2 Pompe children) who with others spearheaded a biotechnology startup Amicus Therapeutics Inc which attempts to develop treatment for genetic diseases. |