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Overcoming Barriers in Pompe Disease Treatment and Research in Eastern Europe
Overcoming Barriers in Pompe Disease Treatment and Research in Eastern Europe Pompe disease is a rare genetic disorder caused by a deficiency of the enzyme acid alpha-glucosidase (GAA), leading to progressive muscle weakness, respiratory complications, and organ damage. While global advancements in Pompe disease treatments are encouraging, Eastern Europe faces unique challenges in diagnosis,...
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