Thrombotic Microangiopathy Following Onasemnogene Abeparvovec for Spinal Muscular Atrophy: A Case Series
Chand, Deepa H., Zaidman, Craig, Arya, Kapil, Millner, Rachel, Farrar, Michelle A., Mackie, Fiona E., Goedeker, Natalie L., Dharnidharka, Vikas R., Dandamudi, Raja, Reyna, Sandra P.
Published in The Journal of pediatrics (01.04.2021)
Published in The Journal of pediatrics (01.04.2021)
Get full text
Journal Article
Emerging therapies and challenges in spinal muscular atrophy
Farrar, Michelle A., Park, Susanna B., Vucic, Steve, Carey, Kate A., Turner, Bradley J., Gillingwater, Thomas H., Swoboda, Kathryn J., Kiernan, Matthew C.
Published in Annals of neurology (01.03.2017)
Published in Annals of neurology (01.03.2017)
Get full text
Journal Article
Integrating Ethics and Equity with Economics and Effectiveness for newborn screening in the genomic age: A qualitative study protocol of stakeholder perspectives
Kariyawasam, Didu S, Scarfe, Joanne, Meagher, Christian, Farrar, Michelle A, Bhattacharya, Kaustav, Carter, Stacy M, Newson, Ainsley J, Otlowski, Margaret, Watson, Jo, Millis, Nicole, Norris, Sarah
Published in PloS one (25.03.2024)
Published in PloS one (25.03.2024)
Get full text
Journal Article
Clinical Characteristics and Functional Motor Outcomes of Enterovirus 71 Neurological Disease in Children
Teoh, Hooi-Ling, Mohammad, Shekeeb S, Britton, Philip N, Kandula, Tejaswi, Lorentzos, Michelle S, Booy, Robert, Jones, Cheryl A, Rawlinson, William, Ramachandran, Vidiya, Rodriguez, Michael L, Andrews, P Ian, Dale, Russell C, Farrar, Michelle A, Sampaio, Hugo
Published in JAMA neurology (01.03.2016)
Published in JAMA neurology (01.03.2016)
Get more information
Journal Article
Health, wellbeing and lived experiences of adults with SMA: a scoping systematic review
Wan, Hamish W Y, Carey, Kate A, D'Silva, Arlene, Vucic, Steve, Kiernan, Matthew C, Kasparian, Nadine A, Farrar, Michelle A
Published in Orphanet journal of rare diseases (12.03.2020)
Published in Orphanet journal of rare diseases (12.03.2020)
Get full text
Journal Article
Pathophysiological Insights Derived by Natural History and Motor Function of Spinal Muscular Atrophy
Farrar, Michelle A., MBBS, Vucic, Steve, MBBS, PhD, Johnston, Heather M., MBChB, du Sart, Desirée, MBBS, PhD, Kiernan, Matthew C., MBBS, PhD, DSc
Published in The Journal of pediatrics (2013)
Published in The Journal of pediatrics (2013)
Get full text
Journal Article
Biomarkers and the Development of a Personalized Medicine Approach in Spinal Muscular Atrophy
Kariyawasam, Didu S T, D'Silva, Arlene, Lin, Cindy, Ryan, Monique M, Farrar, Michelle A
Published in Frontiers in neurology (19.08.2019)
Published in Frontiers in neurology (19.08.2019)
Get full text
Journal Article
Financial, opportunity and psychosocial costs of spinal muscular atrophy: an exploratory qualitative analysis of Australian carer perspectives
Farrar, Michelle A, Carey, Kate A, Paguinto, Sarah-Grace, Chambers, Georgina, Kasparian, Nadine A
Published in BMJ open (01.05.2018)
Published in BMJ open (01.05.2018)
Get full text
Journal Article
The involvement of rare disease patient organisations in therapeutic innovation across rare paediatric neurological conditions: a narrative review
Nguyen, Christina Q, Alba-Concepcion, Kristine, Palmer, Elizabeth E, Scully, Jackie L, Millis, Nicole, Farrar, Michelle A
Published in Orphanet journal of rare diseases (18.04.2022)
Published in Orphanet journal of rare diseases (18.04.2022)
Get full text
Journal Article
"Getting ready for the adult world": how adults with spinal muscular atrophy perceive and experience healthcare, transition and well-being
Wan, Hamish W Y, Carey, Kate A, D'Silva, Arlene, Kasparian, Nadine A, Farrar, Michelle A
Published in Orphanet journal of rare diseases (02.04.2019)
Published in Orphanet journal of rare diseases (02.04.2019)
Get full text
Journal Article
The changing therapeutic landscape of spinal muscular atrophy
Davidson, Joanne E, Farrar, Michelle A
Published in Australian journal of general practice (01.01.2022)
Published in Australian journal of general practice (01.01.2022)
Get full text
Journal Article
The psychosocial impact of childhood dementia on children and their parents: a systematic review
Nevin, Suzanne M, McGill, Brittany C, Kelada, Lauren, Hilton, Gail, Maack, Megan, Elvidge, Kristina L, Farrar, Michelle A, Baynam, Gareth, Katz, Naomi T, Donovan, Leigh, Grattan, Sarah, Signorelli, Christina, Bhattacharya, Kaustuv, Nunn, Kenneth, Wakefield, Claire E
Published in Orphanet journal of rare diseases (07.09.2023)
Published in Orphanet journal of rare diseases (07.09.2023)
Get full text
Journal Article
‘Advocacy groups are the connectors’: Experiences and contributions of rare disease patient organization leaders in advanced neurotherapeutics
Nguyen, Christina Q., Kariyawasam, Didu, Alba‐Concepcion, Kristine, Grattan, Sarah, Hetherington, Kate, Wakefield, Claire E., Woolfenden, Susan, Dale, Russell C., Palmer, Elizabeth E., Farrar, Michelle A.
Published in Health expectations : an international journal of public participation in health care and health policy (01.12.2022)
Published in Health expectations : an international journal of public participation in health care and health policy (01.12.2022)
Get full text
Journal Article
Onasemnogene abeparvovec in spinal muscular atrophy: an Australian experience of safety and efficacy
D'Silva, Arlene M., Holland, Sandra, Kariyawasam, Didu, Herbert, Karen, Barclay, Peter, Cairns, Anita, MacLennan, Suzanna C., Ryan, Monique M., Sampaio, Hugo, Smith, Nicholas, Woodcock, Ian R., Yiu, Eppie M., Alexander, Ian E., Farrar, Michelle A.
Published in Annals of clinical and translational neurology (01.03.2022)
Published in Annals of clinical and translational neurology (01.03.2022)
Get full text
Journal Article
“We needed this”: perspectives of parents and healthcare professionals involved in a pilot newborn screening program for spinal muscular atrophy
Kariyawasam, Didu S.T., D'Silva, Arlene M., Vetsch, Janine, Wakefield, Claire E., Wiley, Veronica, Farrar, Michelle A.
Published in EClinicalMedicine (01.03.2021)
Published in EClinicalMedicine (01.03.2021)
Get full text
Journal Article