'High hopes for treatment': Australian stakeholder perspectives of the clinical translation of advanced neurotherapeutics for rare neurological diseases.

Autor: Nguyen, Christina Q.¹, Kariyawasam, Didu S. T.^1,2, Ngai, Tsz Shun Jason¹, Nguyen, James¹, Alba‐Concepcion, Kristine^1,2, Grattan, Sarah E.^1,2, Palmer, Elizabeth E.^1,3, Hetherington, Kate^1,4, Wakefield, Claire E.^1,4, Dale, Russell C.^2,5, Woolfenden, Sue^5,6,7, Mohammad, Shekeeb^2,5, Farrar, Michelle A.^1,2 m.farrar@unsw.edu.au

Publikováno v: Health Expectations. Jun2024, Vol. 27 Issue 3, p1-15. 15p.

The involvement of rare disease patient organisations in therapeutic innovation across rare paediatric neurological conditions: a narrative review.

Autor: Nguyen, Christina Q.^1,2 (AUTHOR), Alba-Concepcion, Kristine^1,2 (AUTHOR), Palmer, Elizabeth E.^2,3 (AUTHOR), Scully, Jackie L.⁴ (AUTHOR), Millis, Nicole⁵ (AUTHOR), Farrar, Michelle A.^1,2 (AUTHOR) m.farrar@unsw.edu.au

Publikováno v: Orphanet Journal of Rare Diseases. 11/12/2022, Vol. 17 Issue 1, p1-10. 10p.

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Conserved multi-tissue transcriptomic adaptations to exercise training in humans and mice.

Autor: Moore TM; Division of Cardiology, Diabetes, and Hypertension, Department of Medicine, David Geffen School of Medicine University of California, Los Angeles, Los Angeles, CA, USA; Division of Endocrinology, Diabetes, and Hypertension, Department of Medicine, David Geffen School of Medicine University of California, Los Angeles, Los Angeles, CA, USA., Lee S; Department of Transplantation, Institute of Clinical Medicine, Faculty of Medicine, University of Oslo, Oslo, Norway., Olsen T; Department of Nutrition, Institute of Basic Medical Sciences, Faculty of Medicine, University of Oslo, Oslo, Norway., Morselli M; Department of Molecular, Cell and Developmental Biology, University of California Los Angeles, Los Angeles, CA, USA; UCLA-DOE Institute for Genomics and Proteomics, University of California Los Angeles, Los Angeles, CA, USA; Institute for Quantitative and Computational Biosciences - The Collaboratory, University of California, Los Angeles, Los Angeles, CA, USA., Strumwasser AR; Division of Endocrinology, Diabetes, and Hypertension, Department of Medicine, David Geffen School of Medicine University of California, Los Angeles, Los Angeles, CA, USA., Lin AJ; Division of Endocrinology, Diabetes, and Hypertension, Department of Medicine, David Geffen School of Medicine University of California, Los Angeles, Los Angeles, CA, USA; Department of Chemical and Systems Biology, Stanford School of Medicine, Stanford, CA, USA., Zhou Z; Division of Endocrinology, Diabetes, and Hypertension, Department of Medicine, David Geffen School of Medicine University of California, Los Angeles, Los Angeles, CA, USA., Abrishami A; Department of Transplantation, Institute of Clinical Medicine, Faculty of Medicine, University of Oslo, Oslo, Norway., Garcia SM; Division of Endocrinology, Diabetes, and Hypertension, Department of Medicine, David Geffen School of Medicine University of California, Los Angeles, Los Angeles, CA, USA., Bribiesca J; Division of Endocrinology, Diabetes, and Hypertension, Department of Medicine, David Geffen School of Medicine University of California, Los Angeles, Los Angeles, CA, USA., Cory K; Division of Endocrinology, Diabetes, and Hypertension, Department of Medicine, David Geffen School of Medicine University of California, Los Angeles, Los Angeles, CA, USA., Whitney K; Division of Endocrinology, Diabetes, and Hypertension, Department of Medicine, David Geffen School of Medicine University of California, Los Angeles, Los Angeles, CA, USA., Ho T; Division of Endocrinology, Diabetes, and Hypertension, Department of Medicine, David Geffen School of Medicine University of California, Los Angeles, Los Angeles, CA, USA., Ho T; Division of Endocrinology, Diabetes, and Hypertension, Department of Medicine, David Geffen School of Medicine University of California, Los Angeles, Los Angeles, CA, USA., Lee JL; Division of Endocrinology, Diabetes, and Hypertension, Department of Medicine, David Geffen School of Medicine University of California, Los Angeles, Los Angeles, CA, USA., Rucker DH; Division of Endocrinology, Diabetes, and Hypertension, Department of Medicine, David Geffen School of Medicine University of California, Los Angeles, Los Angeles, CA, USA., Nguyen CQA; Division of Endocrinology, Diabetes, and Hypertension, Department of Medicine, David Geffen School of Medicine University of California, Los Angeles, Los Angeles, CA, USA., Anand ATS; Division of Endocrinology, Diabetes, and Hypertension, Department of Medicine, David Geffen School of Medicine University of California, Los Angeles, Los Angeles, CA, USA., Yackly A; Division of Endocrinology, Diabetes, and Hypertension, Department of Medicine, David Geffen School of Medicine University of California, Los Angeles, Los Angeles, CA, USA., Mendoza LQ; Division of Endocrinology, Diabetes, and Hypertension, Department of Medicine, David Geffen School of Medicine University of California, Los Angeles, Los Angeles, CA, USA., Leyva BK; Division of Endocrinology, Diabetes, and Hypertension, Department of Medicine, David Geffen School of Medicine University of California, Los Angeles, Los Angeles, CA, USA., Aliman C; Department of Transplantation, Institute of Clinical Medicine, Faculty of Medicine, University of Oslo, Oslo, Norway., Artiga DJ; Division of Endocrinology, Diabetes, and Hypertension, Department of Medicine, David Geffen School of Medicine University of California, Los Angeles, Los Angeles, CA, USA., Meng Y; Division of Cardiology, Diabetes, and Hypertension, Department of Medicine, David Geffen School of Medicine University of California, Los Angeles, Los Angeles, CA, USA., Charugundla S; Division of Cardiology, Diabetes, and Hypertension, Department of Medicine, David Geffen School of Medicine University of California, Los Angeles, Los Angeles, CA, USA., Pan C; Division of Cardiology, Diabetes, and Hypertension, Department of Medicine, David Geffen School of Medicine University of California, Los Angeles, Los Angeles, CA, USA., Jedian V; Division of Cardiology, Diabetes, and Hypertension, Department of Medicine, David Geffen School of Medicine University of California, Los Angeles, Los Angeles, CA, USA., Seldin MM; Division of Cardiology, Diabetes, and Hypertension, Department of Medicine, David Geffen School of Medicine University of California, Los Angeles, Los Angeles, CA, USA; Department of Biological Chemistry and Center for Epigenetics and Metabolism, University of California, Irvine, Irvine, CA, USA., Ahn IS; Molecular, Cellular, and Integrative Physiology Interdepartmental Program, University of California, Los Angeles, Los Angeles, CA, USA., Diamante G; Molecular, Cellular, and Integrative Physiology Interdepartmental Program, University of California, Los Angeles, Los Angeles, CA, USA., Blencowe M; Molecular, Cellular, and Integrative Physiology Interdepartmental Program, University of California, Los Angeles, Los Angeles, CA, USA; Institute for Quantitative and Computational Biosciences, University of California, Los Angeles, Los Angeles, CA, USA., Yang X; Molecular, Cellular, and Integrative Physiology Interdepartmental Program, University of California, Los Angeles, Los Angeles, CA, USA; Department of Integrative Biology and Physiology, University of California, Los Angeles, Los Angeles, CA, USA; Institute for Quantitative and Computational Biosciences, University of California, Los Angeles, Los Angeles, CA, USA., Mouisel E; Institute of Metabolic and Cardiovascular Diseases, UMR1297 Inserm, Paul Sabatier University, Toulouse, France., Pellegrini M; UCLA-DOE Institute for Genomics and Proteomics, University of California Los Angeles, Los Angeles, CA, USA., Turcotte LP; Department of Biological Sciences, Dana & David Dornsife College of Letters, Arts, and Sciences, University of Southern California, Los Angeles, CA, USA., Birkeland KI; Department of Transplantation, Institute of Clinical Medicine, Faculty of Medicine, University of Oslo, Oslo, Norway., Norheim F; Division of Cardiology, Diabetes, and Hypertension, Department of Medicine, David Geffen School of Medicine University of California, Los Angeles, Los Angeles, CA, USA; Department of Nutrition, Institute of Basic Medical Sciences, Faculty of Medicine, University of Oslo, Oslo, Norway., Drevon CA; Department of Nutrition, Institute of Basic Medical Sciences, Faculty of Medicine, University of Oslo, Oslo, Norway., Lusis AJ; Division of Cardiology, Diabetes, and Hypertension, Department of Medicine, David Geffen School of Medicine University of California, Los Angeles, Los Angeles, CA, USA; Department of Human Genetics, University of California, Los Angeles, Los Angeles, CA, USA; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA, USA., Hevener AL; Division of Endocrinology, Diabetes, and Hypertension, Department of Medicine, David Geffen School of Medicine University of California, Los Angeles, Los Angeles, CA, USA; Iris Cantor-UCLA Women's Health Research Center, Los Angeles, CA, USA; Veterans Administration Greater Los Angeles Healthcare System, Geriatric Research Education and Clinical Center (GRECC), Los Angeles, CA, USA. Electronic address: ahevener@mednet.ucla.edu.

Publikováno v: Cell reports [Cell Rep] 2023 May 30; Vol. 42 (5), pp. 112499. Date of Electronic Publication: 2023 May 12.