Hasil Pencarian - Michelle A. Farrar

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  1. 1
    Circulating neurofilaments to track dorsal root ganglion toxicity risks with AAV-mediated gene therapy

    Circulating neurofilaments to track dorsal root ganglion toxicity risks with AAV-mediated gene therapy oleh Michelle A. Farrar, Ewout Groen, Christiano Alves

    Diterbitkan 2022-09-01
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  2. 2
    Biomarkers and the Development of a Personalized Medicine Approach in Spinal Muscular Atrophy

    Biomarkers and the Development of a Personalized Medicine Approach in Spinal Muscular Atrophy oleh Didu S. T. Kariyawasam, Didu S. T. Kariyawasam, Arlene D'Silva, Cindy Lin, Monique M. Ryan, Michelle A. Farrar, Michelle A. Farrar

    Diterbitkan 2019-08-01
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  3. 3
    Identification of Novel CSF-Derived miRNAs in Treated Paediatric Onset Spinal Muscular Atrophy: An Exploratory Study

    Identification of Novel CSF-Derived miRNAs in Treated Paediatric Onset Spinal Muscular Atrophy: An Exploratory Study oleh Arlene M. D’Silva, Didu Kariyawasam, Pooja Venkat, Chelsea Mayoh, Michelle A. Farrar

    Diterbitkan 2023-01-01
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  4. 4
    Newborn screening for Duchenne muscular dystrophy: the perspectives of stakeholdersResearch in context

    Newborn screening for Duchenne muscular dystrophy: the perspectives of stakeholdersResearch in context oleh Charli Ji, Didu S. Kariyawasam, Hugo Sampaio, Michelle Lorentzos, Kristi J. Jones, Michelle A. Farrar

    Diterbitkan 2024-04-01
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  5. 5
    Modelling the Cost-Effectiveness and Budget Impact of a Newborn Screening Program for Spinal Muscular Atrophy and Severe Combined Immunodeficiency

    Modelling the Cost-Effectiveness and Budget Impact of a Newborn Screening Program for Spinal Muscular Atrophy and Severe Combined Immunodeficiency oleh Sophy T. F. Shih, Elena Keller, Veronica Wiley, Michelle A. Farrar, Melanie Wong, Georgina M. Chambers

    Diterbitkan 2022-07-01
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  6. 6
    Economic Evaluation of Newborn Screening for Severe Combined Immunodeficiency

    Economic Evaluation of Newborn Screening for Severe Combined Immunodeficiency oleh Sophy T. F. Shih, Elena Keller, Veronica Wiley, Melanie Wong, Michelle A. Farrar, Georgina M. Chambers

    Diterbitkan 2022-07-01
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  7. 7
    “The usual challenges of work are all magnified”: Australian paediatric health professionals’ experiences during the COVID-19 pandemic

    “The usual challenges of work are all magnified”: Australian paediatric health professionals’ experiences during the COVID-19 pandemic oleh Farah Akram, Madeleine Pidcock, Diane Oake, Gary F. Sholler, Michelle A. Farrar, Nadine A. Kasparian

    Diterbitkan 2023-03-01
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  8. 8
    “Getting ready for the adult world”: how adults with spinal muscular atrophy perceive and experience healthcare, transition and well-being

    “Getting ready for the adult world”: how adults with spinal muscular atrophy perceive and experience healthcare, transition and well-being oleh Hamish W. Y. Wan, Kate A. Carey, Arlene D’Silva, Nadine A. Kasparian, Michelle A. Farrar

    Diterbitkan 2019-04-01
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  9. 9
    The involvement of rare disease patient organisations in therapeutic innovation across rare paediatric neurological conditions: a narrative review

    The involvement of rare disease patient organisations in therapeutic innovation across rare paediatric neurological conditions: a narrative review oleh Christina Q. Nguyen, Kristine Alba-Concepcion, Elizabeth E. Palmer, Jackie L. Scully, Nicole Millis, Michelle A. Farrar

    Diterbitkan 2022-04-01
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  10. 10
    Clinician Understanding, Acceptance and utilization of Ketogenic diet therapy for epilepsy in Australia and New Zealand: An online survey

    Clinician Understanding, Acceptance and utilization of Ketogenic diet therapy for epilepsy in Australia and New Zealand: An online survey oleh Tania E. Farrar, Arlene D’Silva, Michael Cardamone, Melissa L. Bartley, Chong H. Wong, Michelle A. Farrar

    Diterbitkan 2024-01-01
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  11. 11
    Gene therapy-based strategies for spinal muscular atrophy—an Asia-Pacific perspective

    Gene therapy-based strategies for spinal muscular atrophy—an Asia-Pacific perspective oleh Michelle A. Farrar, Loudella Calotes-Castillo, Ranil De Silva, Peter Barclay, Lani Attwood, Julie Cini, Monica Ferrie, Didu S. Kariyawasam

    Diterbitkan 2023-11-01
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  12. 12
    Health, wellbeing and lived experiences of adults with SMA: a scoping systematic review

    Health, wellbeing and lived experiences of adults with SMA: a scoping systematic review oleh Hamish W. Y. Wan, Kate A. Carey, Arlene D’Silva, Steve Vucic, Matthew C. Kiernan, Nadine A. Kasparian, Michelle A. Farrar

    Diterbitkan 2020-03-01
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  13. 13
    'Integrating Ethics and Equity with Economics and Effectiveness for newborn screening in the genomic age: A qualitative study protocol of stakeholder perspectives.

    'Integrating Ethics and Equity with Economics and Effectiveness for newborn screening in the genomic age: A qualitative study protocol of stakeholder perspectives. oleh Didu S Kariyawasam, Joanne Scarfe, Christian Meagher, Michelle A Farrar, Kaustav Bhattacharya, Stacy M Carter, Ainsley J Newson, Margaret Otlowski, Jo Watson, Nicole Millis, Sarah Norris

    Diterbitkan 2024-01-01
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  14. 14
    ‘Advocacy groups are the connectors’: Experiences and contributions of rare disease patient organization leaders in advanced neurotherapeutics

    ‘Advocacy groups are the connectors’: Experiences and contributions of rare disease patient organization leaders in advanced neurotherapeutics oleh Christina Q. Nguyen, Didu Kariyawasam, Kristine Alba‐Concepcion, Sarah Grattan, Kate Hetherington, Claire E. Wakefield, Susan Woolfenden, Russell C. Dale, Elizabeth E. Palmer, Michelle A. Farrar

    Diterbitkan 2022-12-01
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  15. 15
    “We needed this”: perspectives of parents and healthcare professionals involved in a pilot newborn screening program for spinal muscular atrophy

    “We needed this”: perspectives of parents and healthcare professionals involved in a pilot newborn screening program for spinal muscular atrophy oleh Didu S.T. Kariyawasam, MBBS, Arlene M. D'Silva, PhD, Janine Vetsch, PhD, Claire E. Wakefield, PhD, Veronica Wiley, PhD, Michelle A. Farrar, PhD

    Diterbitkan 2021-03-01
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  16. 16
    The psychosocial impact of childhood dementia on children and their parents: a systematic review

    The psychosocial impact of childhood dementia on children and their parents: a systematic review oleh Suzanne M. Nevin, Brittany C. McGill, Lauren Kelada, Gail Hilton, Megan Maack, Kristina L. Elvidge, Michelle A. Farrar, Gareth Baynam, Naomi T. Katz, Leigh Donovan, Sarah Grattan, Christina Signorelli, Kaustuv Bhattacharya, Kenneth Nunn, Claire E. Wakefield

    Diterbitkan 2023-09-01
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  17. 17
    Onasemnogene abeparvovec in spinal muscular atrophy: an Australian experience of safety and efficacy

    Onasemnogene abeparvovec in spinal muscular atrophy: an Australian experience of safety and efficacy oleh Arlene M. D'Silva, Sandra Holland, Didu Kariyawasam, Karen Herbert, Peter Barclay, Anita Cairns, Suzanna C. MacLennan, Monique M. Ryan, Hugo Sampaio, Nicholas Smith, Ian R. Woodcock, Eppie M. Yiu, Ian E. Alexander, Michelle A. Farrar

    Diterbitkan 2022-03-01
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  18. 18
    Whole Genome Sequencing Identifies a 78 kb Insertion from Chromosome 8 as the Cause of Charcot-Marie-Tooth Neuropathy CMTX3.

    Whole Genome Sequencing Identifies a 78 kb Insertion from Chromosome 8 as the Cause of Charcot-Marie-Tooth Neuropathy CMTX3. oleh Megan H Brewer, Rabia Chaudhry, Jessica Qi, Aditi Kidambi, Alexander P Drew, Manoj P Menezes, Monique M Ryan, Michelle A Farrar, David Mowat, Gopinath M Subramanian, Helen K Young, Stephan Zuchner, Stephen W Reddel, Garth A Nicholson, Marina L Kennerson

    Diterbitkan 2016-07-01
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  19. 19
    A national harmonised data collection network for neurodevelopmental disorders: A transdiagnostic assessment protocol for neurodevelopment, mental health, functioning and well‐being

    A national harmonised data collection network for neurodevelopmental disorders: A transdiagnostic assessment protocol for neurodevelopment, mental health, functioning and well‐bein... oleh Kelsie A. Boulton, David Coghill, Natalie Silove, Elizabeth Pellicano, Andrew J. O. Whitehouse, Mark A. Bellgrove, Nicole J. Rinehart, Suncica Lah, Marie‐Antoinette Redoblado‐Hodge, Nadia Badawi, Helen Heussler, Nicole Rogerson, Joshua Burns, Michelle A. Farrar, Ralph Nanan, Iona Novak, Micah B. Goldwater, Natalie Munro, Leanne Togher, Natasha Nassar, Phillipa Quinn, Christel M. Middeldorp, Adam J. Guastella

    Diterbitkan 2021-12-01
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  20. 20
    CSF neopterin, quinolinic acid and kynurenine/tryptophan ratio are biomarkers of active neuroinflammationResearch in context

    CSF neopterin, quinolinic acid and kynurenine/tryptophan ratio are biomarkers of active neuroinflammationResearch in context oleh Jingya Yan, Kavitha Kothur, Shekeeb Mohammad, Jason Chung, Shrujna Patel, Hannah F. Jones, Brooke A. Keating, Velda X. Han, Richard Webster, Simone Ardern-Holmes, Jayne Antony, Manoj P. Menezes, Esther Tantsis, Deepak Gill, Sachin Gupta, Tejaswi Kandula, Hugo Sampaio, Michelle A. Farrar, Christopher Troedson, P Ian Andrews, Sekhar C. Pillai, Benjamin Heng, Gilles J. Guillemin, Anna Guller, Sushil Bandodkar, Russell C. Dale

    Diterbitkan 2023-05-01
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