Delivery of Neuroregenerative Proteins to the Brain for Treatments of Neurodegenerative Brain Diseases.

Autor: Ebert, Eric T.¹ (AUTHOR) eric.ebert@ku.edu, Schwinghamer, Kelly M.¹ (AUTHOR), Siahaan, Teruna J.¹ (AUTHOR) siahaan@ku.edu

Publikováno v: Life (2075-1729). Nov2024, Vol. 14 Issue 11, p1456. 31p.

Characterization of Fluid Biomarkers Reveals Lysosome Dysfunction and Neurodegeneration in Neuronopathic MPS II Patients.

Autor: Bhalla A; Denali Therapeutics Inc., South San Francisco, CA 94080, USA., Ravi R; Denali Therapeutics Inc., South San Francisco, CA 94080, USA., Fang M; Denali Therapeutics Inc., South San Francisco, CA 94080, USA., Arguello A; Denali Therapeutics Inc., South San Francisco, CA 94080, USA., Davis SS; Denali Therapeutics Inc., South San Francisco, CA 94080, USA., Chiu CL; Denali Therapeutics Inc., South San Francisco, CA 94080, USA., Blumenfeld JR; Denali Therapeutics Inc., South San Francisco, CA 94080, USA.; Department of Neuroscience, University of California, San Francisco, San Francisco, CA 94158, USA., Nguyen HN; Denali Therapeutics Inc., South San Francisco, CA 94080, USA., Earr TK; Denali Therapeutics Inc., South San Francisco, CA 94080, USA., Wang J; Denali Therapeutics Inc., South San Francisco, CA 94080, USA., Astarita G; Denali Therapeutics Inc., South San Francisco, CA 94080, USA., Zhu Y; Denali Therapeutics Inc., South San Francisco, CA 94080, USA., Fiore D; Denali Therapeutics Inc., South San Francisco, CA 94080, USA., Scearce-Levie K; Denali Therapeutics Inc., South San Francisco, CA 94080, USA., Diaz D; Denali Therapeutics Inc., South San Francisco, CA 94080, USA., Cahan H; Denali Therapeutics Inc., South San Francisco, CA 94080, USA., Troyer MD; Denali Therapeutics Inc., South San Francisco, CA 94080, USA., Harris JM; Denali Therapeutics Inc., South San Francisco, CA 94080, USA., Escolar ML; Department of Pediatrics, Children's Hospital of Pittsburgh, Pittsburgh, PA 15224, USA.

Publikováno v: International journal of molecular sciences [Int J Mol Sci] 2020 Jul 22; Vol. 21 (15). Date of Electronic Publication: 2020 Jul 22.

Intracerebroventricular cerliponase alfa for children with CLN2 disease: Interim results from an ongoing phase 2 extension study

Autor: Schulz, A., Specchio, N., Gissen, P., de los Reyes, E., Cahan, H., Slasor, P., Ajayi, T., Jacoby, D.

Publikováno v: In European Journal of Paediatric Neurology June 2017 21 Supplement 1:e21-e21

Study of Intraventricular Cerliponase Alfa for CLN2 Disease.

Autor: Schulz A; From the Department of Pediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany (A.S., A.K.); BioMarin Pharmaceutical, Novato, CA (T.A., H.C., P.S., D.J.); the Department of Neuroscience, Bambino Gesù Children's Hospital, IRCCS, Rome (N.S.); Nationwide Children's Hospital and Ohio State University, Columbus (E.L.R.); UCL Great Ormond Street Institute of Child Health, London (P.G.); and the Citigroup Biomedical Imaging Center, Departments of Radiology and Genetic Medicine, Weill Cornell Medical College, New York (D.B., J.P.D.)., Ajayi T; From the Department of Pediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany (A.S., A.K.); BioMarin Pharmaceutical, Novato, CA (T.A., H.C., P.S., D.J.); the Department of Neuroscience, Bambino Gesù Children's Hospital, IRCCS, Rome (N.S.); Nationwide Children's Hospital and Ohio State University, Columbus (E.L.R.); UCL Great Ormond Street Institute of Child Health, London (P.G.); and the Citigroup Biomedical Imaging Center, Departments of Radiology and Genetic Medicine, Weill Cornell Medical College, New York (D.B., J.P.D.)., Specchio N; From the Department of Pediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany (A.S., A.K.); BioMarin Pharmaceutical, Novato, CA (T.A., H.C., P.S., D.J.); the Department of Neuroscience, Bambino Gesù Children's Hospital, IRCCS, Rome (N.S.); Nationwide Children's Hospital and Ohio State University, Columbus (E.L.R.); UCL Great Ormond Street Institute of Child Health, London (P.G.); and the Citigroup Biomedical Imaging Center, Departments of Radiology and Genetic Medicine, Weill Cornell Medical College, New York (D.B., J.P.D.)., de Los Reyes E; From the Department of Pediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany (A.S., A.K.); BioMarin Pharmaceutical, Novato, CA (T.A., H.C., P.S., D.J.); the Department of Neuroscience, Bambino Gesù Children's Hospital, IRCCS, Rome (N.S.); Nationwide Children's Hospital and Ohio State University, Columbus (E.L.R.); UCL Great Ormond Street Institute of Child Health, London (P.G.); and the Citigroup Biomedical Imaging Center, Departments of Radiology and Genetic Medicine, Weill Cornell Medical College, New York (D.B., J.P.D.)., Gissen P; From the Department of Pediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany (A.S., A.K.); BioMarin Pharmaceutical, Novato, CA (T.A., H.C., P.S., D.J.); the Department of Neuroscience, Bambino Gesù Children's Hospital, IRCCS, Rome (N.S.); Nationwide Children's Hospital and Ohio State University, Columbus (E.L.R.); UCL Great Ormond Street Institute of Child Health, London (P.G.); and the Citigroup Biomedical Imaging Center, Departments of Radiology and Genetic Medicine, Weill Cornell Medical College, New York (D.B., J.P.D.)., Ballon D; From the Department of Pediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany (A.S., A.K.); BioMarin Pharmaceutical, Novato, CA (T.A., H.C., P.S., D.J.); the Department of Neuroscience, Bambino Gesù Children's Hospital, IRCCS, Rome (N.S.); Nationwide Children's Hospital and Ohio State University, Columbus (E.L.R.); UCL Great Ormond Street Institute of Child Health, London (P.G.); and the Citigroup Biomedical Imaging Center, Departments of Radiology and Genetic Medicine, Weill Cornell Medical College, New York (D.B., J.P.D.)., Dyke JP; From the Department of Pediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany (A.S., A.K.); BioMarin Pharmaceutical, Novato, CA (T.A., H.C., P.S., D.J.); the Department of Neuroscience, Bambino Gesù Children's Hospital, IRCCS, Rome (N.S.); Nationwide Children's Hospital and Ohio State University, Columbus (E.L.R.); UCL Great Ormond Street Institute of Child Health, London (P.G.); and the Citigroup Biomedical Imaging Center, Departments of Radiology and Genetic Medicine, Weill Cornell Medical College, New York (D.B., J.P.D.)., Cahan H; From the Department of Pediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany (A.S., A.K.); BioMarin Pharmaceutical, Novato, CA (T.A., H.C., P.S., D.J.); the Department of Neuroscience, Bambino Gesù Children's Hospital, IRCCS, Rome (N.S.); Nationwide Children's Hospital and Ohio State University, Columbus (E.L.R.); UCL Great Ormond Street Institute of Child Health, London (P.G.); and the Citigroup Biomedical Imaging Center, Departments of Radiology and Genetic Medicine, Weill Cornell Medical College, New York (D.B., J.P.D.)., Slasor P; From the Department of Pediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany (A.S., A.K.); BioMarin Pharmaceutical, Novato, CA (T.A., H.C., P.S., D.J.); the Department of Neuroscience, Bambino Gesù Children's Hospital, IRCCS, Rome (N.S.); Nationwide Children's Hospital and Ohio State University, Columbus (E.L.R.); UCL Great Ormond Street Institute of Child Health, London (P.G.); and the Citigroup Biomedical Imaging Center, Departments of Radiology and Genetic Medicine, Weill Cornell Medical College, New York (D.B., J.P.D.)., Jacoby D; From the Department of Pediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany (A.S., A.K.); BioMarin Pharmaceutical, Novato, CA (T.A., H.C., P.S., D.J.); the Department of Neuroscience, Bambino Gesù Children's Hospital, IRCCS, Rome (N.S.); Nationwide Children's Hospital and Ohio State University, Columbus (E.L.R.); UCL Great Ormond Street Institute of Child Health, London (P.G.); and the Citigroup Biomedical Imaging Center, Departments of Radiology and Genetic Medicine, Weill Cornell Medical College, New York (D.B., J.P.D.)., Kohlschütter A; From the Department of Pediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany (A.S., A.K.); BioMarin Pharmaceutical, Novato, CA (T.A., H.C., P.S., D.J.); the Department of Neuroscience, Bambino Gesù Children's Hospital, IRCCS, Rome (N.S.); Nationwide Children's Hospital and Ohio State University, Columbus (E.L.R.); UCL Great Ormond Street Institute of Child Health, London (P.G.); and the Citigroup Biomedical Imaging Center, Departments of Radiology and Genetic Medicine, Weill Cornell Medical College, New York (D.B., J.P.D.).

Publikováno v: The New England journal of medicine [N Engl J Med] 2018 May 17; Vol. 378 (20), pp. 1898-1907. Date of Electronic Publication: 2018 Apr 24.

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