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Pompe Disease

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  • Treating pulmonary dysfunction in Pompe disease with gene therapy

    Researchers at the University of Florida have successfully used gene therapy to treat patients with infantile onset Pompe disease, a progressive condition that severely compromises cardiopulmonary function in the first years of life. The team at UF’s Powell Center for Rare Disease Research and Thera more

  • Intersection between nervous and musculoskeletal system could be key to treating Pompe disease

    Walking and breathing without mechanical assistance typically becomes a struggle for patients who have Pompe disease because of weakened, damaged muscles as they age. Now, University of Florida Health researchers have identified how prior to these devastating effects, hidden physiological changes oc more

  • Oxyrane Raises $26.5m in Series D Financing

    Forbion Capital Partners announced that it has invested in Oxyrane UK Limited, a biopharmaceutical company dedicated to the development of enzyme replacement therapies (ERT) to treat lysosomal storage diseases, a class of more than forty rare inherited diseases. In its oversubscribed Series D financ more

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