Diseases, Conditions, Syndromes

A new treatment for rare muscular disease

Rare diseases are sometimes the most difficult to treat because of a lack of research and fewer participants to study.

Medical research

Study identifies potential new avenue for treating pompe disease

Researchers at Duke Health have identified a potential new avenue for treating Pompe disease, a rare condition caused by the build-up of glycogen, a storage form of sugar, in cardiac and skeletal muscle, the liver and other ...

Genetics

New therapy against rare gene defects

On 15th April is the 1st International Pompe Disease Day, a campaign to raise awareness of this rare but severe gene defect. Pompe Disease is only one of more than 40 metabolic disorders that mainly affect children under ...

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