Search Results - "Palmieri, Kathleen"
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Combined miRNA transcriptome and proteome analysis of extracellular vesicles in urine and blood from the Pompe mouse model
Published in Annals of medicine (Helsinki) (01-12-2024)“…Acid α-glucosidase (GAA) is a lysosomal enzyme that hydrolyzes glycogen to glucose. Deficiency of GAA causes Pompe disease (PD), also known as glycogen storage…”
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Biodistribution of Idursulfase Formulated for Intrathecal Use (Idursulfase-IT) in Cynomolgus Monkeys after Intrathecal Lumbar Administration
Published in PloS one (20-10-2016)“…Enzyme replacement therapy with intravenous idursulfase (recombinant iduronate-2-sulfatase) is approved for the treatment of Hunter syndrome. Intravenous…”
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Journal Article -
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Myostatin and activin blockade by engineered follistatin results in hypertrophy and improves dystrophic pathology in mdx mouse more than myostatin blockade alone
Published in Skeletal muscle (27-10-2018)“…Myostatin antagonists are being developed as therapies for Duchenne muscular dystrophy due to their strong hypertrophic effects on skeletal muscle. Engineered…”
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Gene therapy for cross-correction of somatic organs and the CNS in mucopolysaccharidosis II in rodents and non-human primates
Published in Molecular therapy. Methods & clinical development (08-06-2023)“…Mucopolysaccharidosis II (MPS II) is a rare lysosomal storage disease characterized by deficient activity of iduronate-2-sulfatase (I2S), leading to…”
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Whole Body and CNS Biodistribution of rhHNS in Cynomolgus Monkeys after Intrathecal Lumbar Administration: Treatment Implications for Patients with MPS IIIA
Published in International journal of molecular sciences (01-12-2017)“…Mucopolysaccharidosis III type A (MPS IIIA; Sanfilippo syndrome), a genetic lysosomal disorder causing a deficiency of heparan N-sulfatase (HNS), leads to…”
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Journal Article -
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Biodistribution of Idursulfase Formulated for Intrathecal Use
Published in PloS one (20-10-2016)“…Enzyme replacement therapy with intravenous idursulfase (recombinant iduronate-2-sulfatase) is approved for the treatment of Hunter syndrome. Intravenous…”
Get full text
Journal Article -
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Biodistribution of idursulfase in cynomolgus monkeys after intrathecal-lumbar administration
Published in Molecular genetics and metabolism (01-02-2016)Get full text
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AFTER THE HOUSING BOOM
Published in Bloomberg businessweek (Online) (11-04-2005)“…In today's ownership society, few investments have been so lucrative for so many as homeownership. Since 2001 extremely cheap mortgage rates have fueled a…”
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