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A Phase 3, Open-Label, Extension Study to Assess the Long-term Safety and Efficacy of AVTX-803 in Subjects with Leukocyte Adhesion Deficiency Type II (LAD II)
Rochester, MN
The purpose of this study is to assess the long-term safety and effectiveness of AVTX-803 in subjects with leukocyte adhesion deficiency Type II (LAD II).
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Phase 1/2 Study Of Intravenous Administration Of A Serotype Rh.10 Replication Deficient Adeno-associated Virus Gene Transfer Vector Expressing The Human Propionyl-CoA Carboxylase CDNA To Individuals With Propionic Acidemia
Rochester, MN
The purpose of this study is to evaluate the safety and potential benefit of immunomodulatory gene therapy designed to minimize the immune response.
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Unexplored Pathways: The Impact of Abnormal Glycosylation on the Hypothalamic-Pituitary Adrenal and -Gonadal Axes and Bone Health in Patients with Congenital Disorders of Glycosylation
Rochester, MN
The objectives of this study are to characterize the endocrine phenome of 60 patients with PMM2-CDG through a cross-sectional measurement of endocrine markers of adrenal, thyroid, and gonadal function, bone health, growth and glucose metabolism, along with clinical data and bone density measures, and to establish distinctive glycoproteomic patterns associated with endocrinopathies in PMM2-CDG, and identify biomarkers for adrenal, thyroid, glucose, gonadal, and bone disease.
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