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FDA Approves New Drug for Pompe Disease

The Food and Drug Administration has just approved a new drug to treat those with late-onset Pompe disease. It's called alglucosidase alfa (Lumizyme).

Pompe disease affects between 5,000 and 10,000 people globally. It's a rare lysosomal storage disorder caused by a lack of an enzyme called acid alpha-1,4-glucosidase. This lack leads to a build-up of glycogen, which eventually causes muscle damage, and can be fatal when the respiratory and cardiovascular systems are affected.

Lumizyme replaces the necessary alpha-glucosidase (GAA) in the body, reducing the glycogen buildup. The drug, which was approved based on a trial of 90 patients between 10 and 70 years old, has not been approved for younger patients with Pompe disease. The other effective drug on the market, Myozyme -- which has been approved for younger patients --  is currently in short supply and is being reserved for those with Pompe disease under 8 years old.

Comments
October 13, 2011 at 4:36 am
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i have this sensation that comes underneath my skin, sometimes i feel as it is itching under the skin and sometimes it like i have been burned under the skin or sometime painful. but all these are not on the surface of the skin, its like they are under the skin. i went to so many doctors but i am still without help. i am just wondering if maybe my muscles are not OK. i am seeking for help

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