Highlights
A collection of news and information related to Pompe Disease published by this site and its partners.
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Gene therapy helps muscular dystrophy patients breathe easier
Children with a rare form of muscular dystrophy called Pompe disease often spend their days tethered to mechanical ventilators in order to breathe. But results from a new clinical trial at University of Florida Health show that gene therapy improved...Tags: Science and Technology, Drugs and Medicines, Viral Diseases and Infections, Muscular Dystrophy, Physical Therapy
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Gene therapy may improve Pompe disease symptom
Booster ShotsThe movie "Extraordinary Measures," now in wide release, tells the true story of John and Aileen Crowley, whose two youngest children were diagnosed with Pompe disease. The disease, a form of muscular dystrophy, causes severe muscle weakness and... -
New hope for Pompe Disease patients
A little boy diagnosed with a rare genetic disorder wasn't expected to make it to his first birthday, but a new treatment is helping him not only survive, but thrive. Ira Brown Junior really pays attention to what's going around him. The boy wasn't...Tags: Immune System, Children, Hospitals and Clinics, Health, Health Treatments
Jun 11, 2013
|Story| South Florida Sun-Sentinel
Jan 26, 2010
| Los Angeles Times
Jan 13, 2009
|Story| WXIN-LTV
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