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Akademický článek

RNA Interference Targeting Liver Angiopoietin-Like Protein 3 Protects from Nephrotic Syndrome in a Rat Model Via Amelioration of Pathologic Hypertriglyceridemia.

Autor: Zhao Y; Division of Nephrology and Hypertension, University of California, Irvine, California (Y.Z., M.G., N.D.V., M.K., H.L., N.F., H.M.); Long Beach Memorial Pathology Group, Long Beach, California (E.K.); Department of Pathology and Laboratory Medicine, University of California, Irvine, California (T.F.); Tibor Rubin VA Medical Center, Department of Medicine, Nephrology Section, Long Beach, California (H.M.); and Alnylam Pharmaceuticals Inc, Cambridge, Massachusetts (P.A.H., M.M.S.)., Goto M; Division of Nephrology and Hypertension, University of California, Irvine, California (Y.Z., M.G., N.D.V., M.K., H.L., N.F., H.M.); Long Beach Memorial Pathology Group, Long Beach, California (E.K.); Department of Pathology and Laboratory Medicine, University of California, Irvine, California (T.F.); Tibor Rubin VA Medical Center, Department of Medicine, Nephrology Section, Long Beach, California (H.M.); and Alnylam Pharmaceuticals Inc, Cambridge, Massachusetts (P.A.H., M.M.S.)., Vaziri ND; Division of Nephrology and Hypertension, University of California, Irvine, California (Y.Z., M.G., N.D.V., M.K., H.L., N.F., H.M.); Long Beach Memorial Pathology Group, Long Beach, California (E.K.); Department of Pathology and Laboratory Medicine, University of California, Irvine, California (T.F.); Tibor Rubin VA Medical Center, Department of Medicine, Nephrology Section, Long Beach, California (H.M.); and Alnylam Pharmaceuticals Inc, Cambridge, Massachusetts (P.A.H., M.M.S.)., Khazaeli M; Division of Nephrology and Hypertension, University of California, Irvine, California (Y.Z., M.G., N.D.V., M.K., H.L., N.F., H.M.); Long Beach Memorial Pathology Group, Long Beach, California (E.K.); Department of Pathology and Laboratory Medicine, University of California, Irvine, California (T.F.); Tibor Rubin VA Medical Center, Department of Medicine, Nephrology Section, Long Beach, California (H.M.); and Alnylam Pharmaceuticals Inc, Cambridge, Massachusetts (P.A.H., M.M.S.)., Liu H; Division of Nephrology and Hypertension, University of California, Irvine, California (Y.Z., M.G., N.D.V., M.K., H.L., N.F., H.M.); Long Beach Memorial Pathology Group, Long Beach, California (E.K.); Department of Pathology and Laboratory Medicine, University of California, Irvine, California (T.F.); Tibor Rubin VA Medical Center, Department of Medicine, Nephrology Section, Long Beach, California (H.M.); and Alnylam Pharmaceuticals Inc, Cambridge, Massachusetts (P.A.H., M.M.S.)., Farahanchi N; Division of Nephrology and Hypertension, University of California, Irvine, California (Y.Z., M.G., N.D.V., M.K., H.L., N.F., H.M.); Long Beach Memorial Pathology Group, Long Beach, California (E.K.); Department of Pathology and Laboratory Medicine, University of California, Irvine, California (T.F.); Tibor Rubin VA Medical Center, Department of Medicine, Nephrology Section, Long Beach, California (H.M.); and Alnylam Pharmaceuticals Inc, Cambridge, Massachusetts (P.A.H., M.M.S.)., Khanifar E; Division of Nephrology and Hypertension, University of California, Irvine, California (Y.Z., M.G., N.D.V., M.K., H.L., N.F., H.M.); Long Beach Memorial Pathology Group, Long Beach, California (E.K.); Department of Pathology and Laboratory Medicine, University of California, Irvine, California (T.F.); Tibor Rubin VA Medical Center, Department of Medicine, Nephrology Section, Long Beach, California (H.M.); and Alnylam Pharmaceuticals Inc, Cambridge, Massachusetts (P.A.H., M.M.S.)., Farzaneh T; Division of Nephrology and Hypertension, University of California, Irvine, California (Y.Z., M.G., N.D.V., M.K., H.L., N.F., H.M.); Long Beach Memorial Pathology Group, Long Beach, California (E.K.); Department of Pathology and Laboratory Medicine, University of California, Irvine, California (T.F.); Tibor Rubin VA Medical Center, Department of Medicine, Nephrology Section, Long Beach, California (H.M.); and Alnylam Pharmaceuticals Inc, Cambridge, Massachusetts (P.A.H., M.M.S.)., Haslett PA; Division of Nephrology and Hypertension, University of California, Irvine, California (Y.Z., M.G., N.D.V., M.K., H.L., N.F., H.M.); Long Beach Memorial Pathology Group, Long Beach, California (E.K.); Department of Pathology and Laboratory Medicine, University of California, Irvine, California (T.F.); Tibor Rubin VA Medical Center, Department of Medicine, Nephrology Section, Long Beach, California (H.M.); and Alnylam Pharmaceuticals Inc, Cambridge, Massachusetts (P.A.H., M.M.S.)., Moradi H; Division of Nephrology and Hypertension, University of California, Irvine, California (Y.Z., M.G., N.D.V., M.K., H.L., N.F., H.M.); Long Beach Memorial Pathology Group, Long Beach, California (E.K.); Department of Pathology and Laboratory Medicine, University of California, Irvine, California (T.F.); Tibor Rubin VA Medical Center, Department of Medicine, Nephrology Section, Long Beach, California (H.M.); and Alnylam Pharmaceuticals Inc, Cambridge, Massachusetts (P.A.H., M.M.S.)., Soundarapandian MM; Division of Nephrology and Hypertension, University of California, Irvine, California (Y.Z., M.G., N.D.V., M.K., H.L., N.F., H.M.); Long Beach Memorial Pathology Group, Long Beach, California (E.K.); Department of Pathology and Laboratory Medicine, University of California, Irvine, California (T.F.); Tibor Rubin VA Medical Center, Department of Medicine, Nephrology Section, Long Beach, California (H.M.); and Alnylam Pharmaceuticals Inc, Cambridge, Massachusetts (P.A.H., M.M.S.) msoundar@alnylam.com.

Publikováno v: The Journal of pharmacology and experimental therapeutics [J Pharmacol Exp Ther] 2021 Mar; Vol. 376 (3), pp. 428-435. Date of Electronic Publication: 2020 Dec 18.

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Akademický článek

Activation of Protein Kinase A (PKA) signaling mitigates congenital hyperinsulinism associated hypoglycemia in the Sur1-/- mouse model.

Autor: Soundarapandian MM; Alnylam Pharmaceuticals, Cambridge, Massachusetts, United States of America., Juliana CA; Division of Endocrinology and Diabetes, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States of America., Chai J; Division of Endocrinology and Diabetes, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States of America., Haslett PA; Alnylam Pharmaceuticals, Cambridge, Massachusetts, United States of America., Fitzgerald K; Alnylam Pharmaceuticals, Cambridge, Massachusetts, United States of America., De León DD; Division of Endocrinology and Diabetes, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States of America.; Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, United States of America.

Publikováno v: PloS one [PLoS One] 2020 Jul 31; Vol. 15 (7), pp. e0236892. Date of Electronic Publication: 2020 Jul 31 (Print Publication: 2020).

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Akademický článek

A novel LC-MS/MS assay for heparan sulfate screening in the cerebrospinal fluid of mucopolysaccharidosis IIIA patients.

Autor: Naimy H; Shire, 300 Shire Way, Lexington, MA 02421, USA., Powell KD; Tandem Labs - RTP, A LabCorp Company, 2202 Ellis Road, Suite D, Durham, NC 27703, USA., Moriarity JR; Tandem Labs - RTP, A LabCorp Company, 2202 Ellis Road, Suite D, Durham, NC 27703, USA., Wu J; Shire, 300 Shire Way, Lexington, MA 02421, USA., McCauley TG; Shire, 300 Shire Way, Lexington, MA 02421, USA., Haslett PA; Shire, 300 Shire Way, Lexington, MA 02421, USA.; Current address: Agios Pharmaceuticals, 88 Sidney Street, Cambridge, MA 02139, USA., Barbier AJ; Shire, 300 Shire Way, Lexington, MA 02421, USA.; Current address: Alnylam Pharmaceuticals, Inc, 300 Third Street, Cambridge, MA 02142, USA., Qiu Y; Shire, 300 Shire Way, Lexington, MA 02421, USA.

Publikováno v: Bioanalysis [Bioanalysis] 2016 Feb; Vol. 8 (4), pp. 285-95. Date of Electronic Publication: 2016 Feb 05.

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Akademický článek

Quantifying behaviors of children with Sanfilippo syndrome: the Sanfilippo Behavior Rating Scale.

Autor: Shapiro EG; University of Minnesota, Department of Pediatrics, Minneapolis, MN, USA. Electronic address: shapi004@umn.edu., Nestrasil I; University of Minnesota, Department of Pediatrics, Minneapolis, MN, USA., Ahmed A; University of Minnesota, Department of Pediatrics, Minneapolis, MN, USA., Wey A; University of Minnesota, Division of Biostatistics, Minneapolis, MN, USA., Rudser KR; University of Minnesota, Division of Biostatistics, Minneapolis, MN, USA., Delaney KA; University of Minnesota, Department of Pediatrics, Minneapolis, MN, USA., Rumsey RK; University of Minnesota, Department of Pediatrics, Minneapolis, MN, USA., Haslett PA; Shire, Lexington, MA,USA(1)., Whitley CB; University of Minnesota, Department of Pediatrics, Minneapolis, MN, USA., Potegal M; University of Minnesota, Department of Pediatrics, Minneapolis, MN, USA.

Publikováno v: Molecular genetics and metabolism [Mol Genet Metab] 2015 Apr; Vol. 114 (4), pp. 594-8. Date of Electronic Publication: 2015 Mar 05.

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