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- The defect results in a build-up of glycogen in the lysosome, a saclike storage organelle in the cell that acts as a waste-disposal system, leading to muscle weakness, organ damage including the brain, and possible death.
www.scientificamerican.com › article › pompe-diseaseAsk the Experts: What Is Pompe Disease? | Scientific American
Jan 30, 2019 · Amicus CEO John Crowley has spent 20 years working on developing a therapy that might cure Pompe disease, which affects both of his children.
- Chaperone Therapy for Pompe Disease | Pompe Disease News
People with Pompe disease have a defective acid...
- New Pompe disease treatment 'exciting,' Amicus chairman says
Bringing a third medication for late-onset Pompe disease...
- Chaperone Therapy for Pompe Disease | Pompe Disease News
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Sep 21, 2023 · Pompe disease is classified into two subtypes: infantile-onset Pompe disease (IOPD) and late-onset Pompe disease (LOPD). In IOPD, symptoms typically appear during the first year of life and tend to progress very quickly.
John's involvement with biotechnology started in 1998 with the diagnosis of two of his children with Pompe Disease, a rare, severe, and often fatal neuromuscular disorder.
Nov 9, 2023 · Bringing a third medication for late-onset Pompe disease (LOPD) to the treatment table is a personal triumph for Amicus Therapeutics executive chairman John Crowley.
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Oct 9, 2023 · John Crowley’s role in creating a medicine that helped his two young children survive after being diagnosed with Pompe disease was so poignant that it was dramatized in a Hollywood movie, Extraordinary Measures. The story that came after that was even more dramatic.
- Steve Usdin
Jan 22, 2010 · Extraordinary Measures, a film starring Harrison Ford and Brendan Fraser, hits theaters today. It tells the story of John Crowley and his “race to find a cure for Pompe disease, a rare muscle disorder that threatened to kill two of his three children.”.
Jan 25, 2010 · Health. Inspired by biotechnology executive John Crowley's efforts to save his children, the movie Extraordinary Measures shows basic science and biotechnology teaming up to help researchers...
