July 24, 2005
Ventilation Helps Eight With Pompe’s Disease
German researchers reported in the April 26 issue of Neurology that noninvasive ventilation (NIV) was decidedly helpful in acid maltase deficiency (AMD, or Pompe’s disease) in the eight patients they studied.
Posted by Michelle at 12:59 PM | Comments (0) | TrackBack
June 30, 2005
Gene Therapy Aids Mice With Pompe’s Disease
A team including MDA grantees Andrea Amalfitano, Yuan-Tsong Chen and Dwight Koeberl at Duke University in Durham, N.C., has given mice with acid maltase deficiency (Pompe’s disease) a successful gene-based treatment to compensate for their missing acid maltase enzyme. The finding was announced in the January issue of Molecular Therapy.
Posted by Michelle at 11:55 PM | Comments (0) | TrackBack
June 29, 2005
A courageous kid with a debilitating disease
Lucus was diagnosed with Pompe's Disease, which is extremely rare. It's a metabolic disease where glycogen fills his muscles and kills the cells -- making Luke extremely weak. The prognoses was very poor, doctors said he wouldn't live to see his first birthday.
Well, next month Luke will be 12 and he's in a clinical trial for enzyme replacement therapy which is going very well.
Posted by Michelle at 04:28 PM | Comments (0) | TrackBack
May 24, 2005
Transgenic first from pharming
Mr Pinto embarked on a restructuring, selling off the company's manufacturing operations and an experimental treatment for the rare Pompe's disease that had been its lead product. He slashed the number of projects and reduced head count. The company remains lossmaking, but has almost Euros 30m (Dollars 39m) in cash and has cleared its debt.
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