Innovative Approaches to Overcoming Challenges in Pompe Disease Treatment
Pompe disease is a rare, inherited disorder caused by the deficiency of an enzyme called acid alpha-glucosidase (GAA), which is essential for breaking down glycogen in the body. This results in the accumulation of glycogen in various tissues, particularly the muscles, leading to progressive muscle weakness, respiratory difficulties, and in severe cases, heart failure. While there have...
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