Search Results 1-10 of 16720 for Pompe Disease
People with Pompe disease are not able to make enough of this enzyme. This medicine is to be given only by or under the direct supervision of your doctor. This ...
Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and ...
People with Pompe disease are not able to make enough of this enzyme. This medicine is to be given only by or under the direct supervision of your doctor ...
Miglustat is used in combination with cipaglucosidase alfa-atga injection (Pombiliti™) to treat late-onset of Pompe disease in patients who are not improving on ...
This childhood condition occurs when blood supply to the ball part of the hip joint is interrupted for a short time, making the bone fracture easily and ...
2025 - present. MemberAmerican College of Medical Genetics and Genomics - Therapeutics Committee · 2024 - present. MemberMinnesota Pompe Disease Consortium · 2024 ...
POEMS syndrome is a rare blood disorder that damages nerves and affects other parts of the body. POEMS stands for symptoms of the condition. Symptoms may ...
Type 3 Gaucher disease may be treated with enzyme replacement therapy. This treatment doesn't affect disease progression in the brain. Bone marrow transplants ...
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