Using the ALSFRS-R in multicentre clinical trials for amyotrophic lateral sclerosis: potential limitations in current standard operating procedures

Autor: Jaap N.E. Bakers, Adriaan D. de Jongh, Tommy M. Bunte, Lindsay Kendall, Steve S. Han, Noam Epstein, Arseniy Lavrov, Anita Beelen, Johanna M.A. Visser-Meily, Leonard H. van den Berg, Ruben P. A. van Eijk

Publikováno v: Amyotrophic lateral sclerosisfrontotemporal degeneration. 23(7-8)

iObjective:/iUniform data collection is fundamental for multicentre clinical trials. We aim to determine the variability, between ALS trial centers, in the prevalence of unexpected or implausible improvements in the revised ALS functional rating scal

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::5887c8b967e8e7fa1aec9e4310201d2f
https://pubmed.ncbi.nlm.nih.gov/34949141

Plasma neurofilament light levels show elevation two years prior to diagnosis of amyotrophic lateral sclerosis in the UK Biobank.

Autor: Smith EN; Human Genetics and Systems Biology, Takeda Development Center Americas, Inc. San Diego, CA, USA., Lee J; Human Genetics and Systems Biology, Takeda Development Center Americas, Inc. Cambridge, MA, USA., Prilutsky D; Human Genetics and Systems Biology, Takeda Development Center Americas, Inc. Cambridge, MA, USA., Zicha S; Neuroscience Translational Medicine, Takeda Development Center Americas, Inc. Cambridge, MA, USA, and., Wang Z; Neuroscience Translational Medicine, Takeda Development Center Americas, Inc. Cambridge, MA, USA, and., Han S; Neuroscience Therapeutic Area Unit, Takeda Development Center Americas, Inc. Cambridge, MA, USA., Zach N; Neuroscience Translational Medicine, Takeda Development Center Americas, Inc. Cambridge, MA, USA, and.

Publikováno v: Amyotrophic lateral sclerosis & frontotemporal degeneration [Amyotroph Lateral Scler Frontotemporal Degener] 2024 Feb; Vol. 25 (1-2), pp. 170-176. Date of Electronic Publication: 2024 Jan 23.

Electrical impedance myography detects dystrophin-related muscle changes in mdx mice.

Autor: Hiyoshi T; Neuroscience Translational Medicine, Neuroscience Drug Discovery Unit, Takeda Pharmaceutical Company Limited, 26-1, Muraoka-Higashi 2-Chome, Fujisawa, Kanagawa, 251-8555, Japan., Zhao F; Center of Excellence for Imaging, Preclinical and Translational Sciences, Takeda Development Center Americas, Inc., 95 Hayden Avenue, Lexington, MA, 02141, USA., Baba R; Muscular Disease and Neuropathy Unit, Neuroscience Drug Discovery Unit, Takeda Pharmaceutical Company Limited, 26-1, Muraoka-Higashi 2-Chome, Fujisawa, Kanagawa, 251-8555, Japan., Hirakawa T; Muscular Disease and Neuropathy Unit, Neuroscience Drug Discovery Unit, Takeda Pharmaceutical Company Limited, 26-1, Muraoka-Higashi 2-Chome, Fujisawa, Kanagawa, 251-8555, Japan., Kuboki R; Muscular Disease and Neuropathy Unit, Neuroscience Drug Discovery Unit, Takeda Pharmaceutical Company Limited, 26-1, Muraoka-Higashi 2-Chome, Fujisawa, Kanagawa, 251-8555, Japan., Suzuki K; Muscular Disease and Neuropathy Unit, Neuroscience Drug Discovery Unit, Takeda Pharmaceutical Company Limited, 26-1, Muraoka-Higashi 2-Chome, Fujisawa, Kanagawa, 251-8555, Japan., Tomimatsu Y; Neuroscience Translational Medicine, Neuroscience Drug Discovery Unit, Takeda Pharmaceutical Company Limited, 26-1, Muraoka-Higashi 2-Chome, Fujisawa, Kanagawa, 251-8555, Japan., O'Donnell P; Neuroscience Translational Medicine, Neuroscience Drug Discovery Unit, Takeda Development Center Americas, Inc., 95 Hayden Avenue, Lexington, MA, 02141, USA., Han S; Neuroscience Therapeutic Area Unit, Takeda Development Center Americas, Inc., 95 Hayden Avenue, Lexington, MA, 02141, USA., Zach N; Neuroscience Translational Medicine, Neuroscience Drug Discovery Unit, Takeda Development Center Americas, Inc., 95 Hayden Avenue, Lexington, MA, 02141, USA., Nakashima M; Neuroscience Translational Medicine, Neuroscience Drug Discovery Unit, Takeda Pharmaceutical Company Limited, 26-1, Muraoka-Higashi 2-Chome, Fujisawa, Kanagawa, 251-8555, Japan. masato.nakashima@takeda.com.

Publikováno v: Skeletal muscle [Skelet Muscle] 2023 Nov 18; Vol. 13 (1), pp. 19. Date of Electronic Publication: 2023 Nov 18.

Qualitative measures that assess functional disability and quality of life in ALS.

Autor: Hartmaier SL; CERobs Consulting, LLC, Wrightsville Beach, NC, USA. drsusan@bhstrategy.com., Rhodes T; CERobs Consulting, LLC, Wrightsville Beach, NC, USA., Cook SF; CERobs Consulting, LLC, Wrightsville Beach, NC, USA., Schlusser C; CERobs Consulting, LLC, Wrightsville Beach, NC, USA.; Gillings School of Public Health, University of North Carolina-Chapel Hill, Chapel Hill, NC, USA., Chen C; Takeda Development Center Americas, Inc., Cambridge, MA, USA., Han S; Takeda Development Center Americas, Inc., Cambridge, MA, USA., Zach N; Takeda Development Center Americas, Inc., Cambridge, MA, USA., Murthy V; Takeda Development Center Americas, Inc., Cambridge, MA, USA., Davé S; Takeda Development Center Americas, Inc., Cambridge, MA, USA.

Publikováno v: Health and quality of life outcomes [Health Qual Life Outcomes] 2022 Jan 21; Vol. 20 (1), pp. 12. Date of Electronic Publication: 2022 Jan 21.

Pathways disrupted in human ALS motor neurons identified through genetic correction of mutant SOD1.

Autor: Kiskinis E; The Howard Hughes Medical Institute, USA; Harvard Stem Cell Institute, Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA 02138, USA; Stanley Center for Psychiatric Research, Broad Institute of Harvard and MIT, Cambridge, MA 02138, USA., Sandoe J; The Howard Hughes Medical Institute, USA; Harvard Stem Cell Institute, Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA 02138, USA; Stanley Center for Psychiatric Research, Broad Institute of Harvard and MIT, Cambridge, MA 02138, USA., Williams LA; The Howard Hughes Medical Institute, USA; Harvard Stem Cell Institute, Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA 02138, USA; Stanley Center for Psychiatric Research, Broad Institute of Harvard and MIT, Cambridge, MA 02138, USA., Boulting GL; The Howard Hughes Medical Institute, USA; Harvard Stem Cell Institute, Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA 02138, USA., Moccia R; The Howard Hughes Medical Institute, USA; Harvard Stem Cell Institute, Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA 02138, USA; Stanley Center for Psychiatric Research, Broad Institute of Harvard and MIT, Cambridge, MA 02138, USA., Wainger BJ; FM Kirby Neurobiology Center, Boston Children's Hospital and Department of Neurobiology, Harvard Medical School, Boston, MA 02115, USA; Department of Anesthesia, Critical Care and Pain Medicine, Massachusetts General Hospital, Boston, MA 02115, USA., Han S; The Howard Hughes Medical Institute, USA; Harvard Stem Cell Institute, Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA 02138, USA; Stanley Center for Psychiatric Research, Broad Institute of Harvard and MIT, Cambridge, MA 02138, USA., Peng T; The Howard Hughes Medical Institute, USA; Harvard Stem Cell Institute, Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA 02138, USA; Stanley Center for Psychiatric Research, Broad Institute of Harvard and MIT, Cambridge, MA 02138, USA., Thams S; Project A.L.S./Jenifer Estess Laboratory for Stem Cell Research, Departments of Pathology, Neurology and Neuroscience, Columbia University, Center for Motor Neuron Biology and Disease (MNC), and Columbia Stem Cell Initiative (CSCI), New York, NY 10027, USA., Mikkilineni S; The Howard Hughes Medical Institute, USA; Harvard Stem Cell Institute, Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA 02138, USA; Stanley Center for Psychiatric Research, Broad Institute of Harvard and MIT, Cambridge, MA 02138, USA., Mellin C; FM Kirby Neurobiology Center, Boston Children's Hospital and Department of Neurobiology, Harvard Medical School, Boston, MA 02115, USA., Merkle FT; The Howard Hughes Medical Institute, USA; Harvard Stem Cell Institute, Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA 02138, USA; Stanley Center for Psychiatric Research, Broad Institute of Harvard and MIT, Cambridge, MA 02138, USA., Davis-Dusenbery BN; The Howard Hughes Medical Institute, USA; Harvard Stem Cell Institute, Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA 02138, USA; Stanley Center for Psychiatric Research, Broad Institute of Harvard and MIT, Cambridge, MA 02138, USA., Ziller M; Harvard Stem Cell Institute, Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA 02138, USA., Oakley D; Project A.L.S./Jenifer Estess Laboratory for Stem Cell Research, Departments of Pathology, Neurology and Neuroscience, Columbia University, Center for Motor Neuron Biology and Disease (MNC), and Columbia Stem Cell Initiative (CSCI), New York, NY 10027, USA., Ichida J; The Howard Hughes Medical Institute, USA; Harvard Stem Cell Institute, Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA 02138, USA., Di Costanzo S; The Howard Hughes Medical Institute, USA; Harvard Stem Cell Institute, Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA 02138, USA., Atwater N; The Howard Hughes Medical Institute, USA; Harvard Stem Cell Institute, Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA 02138, USA; Stanley Center for Psychiatric Research, Broad Institute of Harvard and MIT, Cambridge, MA 02138, USA., Maeder ML; Molecular Pathology Unit, Center for Computational and Integrative Biology, and Center for Cancer Research, Massachusetts General Hospital, Charlestown, MA 02129, USA., Goodwin MJ; Molecular Pathology Unit, Center for Computational and Integrative Biology, and Center for Cancer Research, Massachusetts General Hospital, Charlestown, MA 02129, USA., Nemesh J; Stanley Center for Psychiatric Research, Broad Institute of Harvard and MIT, Cambridge, MA 02138, USA; Department of Neurology, University of Massachusetts Medical School, Worcester, MA 01655, USA; Department of Genetics, Harvard Medical School, Boston, MA 02115, USA., Handsaker RE; Stanley Center for Psychiatric Research, Broad Institute of Harvard and MIT, Cambridge, MA 02138, USA; Department of Neurology, University of Massachusetts Medical School, Worcester, MA 01655, USA; Department of Genetics, Harvard Medical School, Boston, MA 02115, USA., Paull D; The New York Stem Cell Foundation Research Institute, New York, NY 10023, USA., Noggle S; The New York Stem Cell Foundation Research Institute, New York, NY 10023, USA., McCarroll SA; Stanley Center for Psychiatric Research, Broad Institute of Harvard and MIT, Cambridge, MA 02138, USA; Department of Neurology, University of Massachusetts Medical School, Worcester, MA 01655, USA; Department of Genetics, Harvard Medical School, Boston, MA 02115, USA., Joung JK; Molecular Pathology Unit, Center for Computational and Integrative Biology, and Center for Cancer Research, Massachusetts General Hospital, Charlestown, MA 02129, USA., Woolf CJ; FM Kirby Neurobiology Center, Boston Children's Hospital and Department of Neurobiology, Harvard Medical School, Boston, MA 02115, USA., Brown RH; Department of Neurology, University of Massachusetts Medical School, Worcester, MA 01655, USA., Eggan K; The Howard Hughes Medical Institute, USA; Harvard Stem Cell Institute, Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA 02138, USA; Stanley Center for Psychiatric Research, Broad Institute of Harvard and MIT, Cambridge, MA 02138, USA. Electronic address: eggan@mcb.harvard.edu.

Publikováno v: Cell stem cell [Cell Stem Cell] 2014 Jun 05; Vol. 14 (6), pp. 781-95. Date of Electronic Publication: 2014 Apr 03.