AB-1009 is an investigational gene therapy that has not been approved by any regulatory authority, and its efficacy and safety have not been established or fully evaluated. For more information, ...
Pompe disease is a rare metabolic disorder. It’s marked by a deficiency in lysosomal alpha-glucosidase. Your body needs this enzyme to break down stored glucose, which is called glycogen. When your ...
Pompe disease is a rare genetic condition that affects only 1 person in every 40,000 people in the United States, according to the National Institute of Neurological Disorders and Stroke (NINDS).
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