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A Simple Guide to Pompe Disease (Glycogen Storage Disease II), Diagnosis, Treatment and Related Conditions

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This book describes Pompe Disease, Diagnosis and Treatment and Related Diseases

The film Extraordinary Measures is based on the true story of John and Aileen Crowley, whose children have Pompe disease.
By the year 2000, with the help of a scientist friend, John Crowley raised enough money to start a small biotech company in the hopes of finding a cure for Pompe disease.
His company helped to develop Myozyme, a drug used for treating Pompe disease, which became the first generation therapy that...