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

por Farrar, Michelle A., Groen, Ewout, Alves, Christiano R.R.
Publicado 2022

Oculomotor Apraxia as an Early Presenting Sign of Juvenile‐Onset Huntington's Disease

por Innes, Emily Amy, Qiu, Jessica, Morales‐Briceño, Hugo, Farrar, Michelle A., Mohammad, Shekeeb S.
Publicado 2023

Biomarkers and the Development of a Personalized Medicine Approach in Spinal Muscular Atrophy

por Kariyawasam, Didu S. T., D'Silva, Arlene, Lin, Cindy, Ryan, Monique M., Farrar, Michelle A.
Publicado 2019

Integrating newborn screening for spinal muscular atrophy into health care systems: an Australian pilot programme

por D'Silva, Arlene M, Kariyawasam, Didu S T, Best, Stephanie, Wiley, Veronica, Farrar, Michelle A
Publicado 2021

Identification of Novel CSF-Derived miRNAs in Treated Paediatric Onset Spinal Muscular Atrophy: An Exploratory Study

por D’Silva, Arlene M., Kariyawasam, Didu, Venkat, Pooja, Mayoh, Chelsea, Farrar, Michelle A.
Publicado 2023

Burning pain: axonal dysfunction in erythromelalgia

por Farrar, Michelle A., Lee, Ming-Jen, Howells, James, Andrews, Peter I., Lin, Cindy S.-Y.
Publicado 2017

Financial, opportunity and psychosocial costs of spinal muscular atrophy: an exploratory qualitative analysis of Australian carer perspectives

por Farrar, Michelle A, Carey, Kate A, Paguinto, Sarah-Grace, Chambers, Georgina, Kasparian, Nadine A
Publicado 2018

Economic Evaluation of Newborn Screening for Severe Combined Immunodeficiency

por Shih, Sophy T. F., Keller, Elena, Wiley, Veronica, Wong, Melanie, Farrar, Michelle A., Chambers, Georgina M.
Publicado 2022

Modelling the Cost-Effectiveness and Budget Impact of a Newborn Screening Program for Spinal Muscular Atrophy and Severe Combined Immunodeficiency

por Shih, Sophy T. F., Keller, Elena, Wiley, Veronica, Farrar, Michelle A., Wong, Melanie, Chambers, Georgina M.
Publicado 2022

“The usual challenges of work are all magnified”: Australian paediatric health professionals’ experiences during the COVID-19 pandemic

por Akram, Farah, Pidcock, Madeleine, Oake, Diane, Sholler, Gary F., Farrar, Michelle A., Kasparian, Nadine A.
Publicado 2023

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

por Wan, Hamish W. Y., Carey, Kate A., D’Silva, Arlene, Kasparian, Nadine A., Farrar, Michelle A.
Publicado 2019

“We needed this”: perspectives of parents and healthcare professionals involved in a pilot newborn screening program for spinal muscular atrophy

por Kariyawasam, Didu S.T., D'Silva, Arlene M., Vetsch, Janine, Wakefield, Claire E., Wiley, Veronica, Farrar, Michelle A.
Publicado 2021

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

por Nguyen, Christina Q., Alba-Concepcion, Kristine, Palmer, Elizabeth E., Scully, Jackie L., Millis, Nicole, Farrar, Michelle A.
Publicado 2022

Gene therapy-based strategies for spinal muscular atrophy—an Asia-Pacific perspective

por Farrar, Michelle A., Calotes-Castillo, Loudella, De Silva, Ranil, Barclay, Peter, Attwood, Lani, Cini, Julie, Ferrie, Monica, Kariyawasam, Didu S.
Publicado 2023

The collective burden of childhood dementia: a scoping review

por Elvidge, Kristina L, Christodoulou, John, Farrar, Michelle A, Tilden, Dominic, Maack, Megan, Valeri, Madeline, Ellis, Magda, Smith, Nicholas J C
Publicado 2023

An Integrated Safety Analysis of Infants and Children with Symptomatic Spinal Muscular Atrophy (SMA) Treated with Nusinersen in Seven Clinical Trials

por Darras, Basil T., Farrar, Michelle A., Mercuri, Eugenio, Finkel, Richard S., Foster, Richard, Hughes, Steven G., Bhan, Ishir, Farwell, Wildon, Gheuens, Sarah
Publicado 2019

Expert recommendations and clinical considerations in the use of onasemnogene abeparvovec gene therapy for spinal muscular atrophy

por Kichula, Elizabeth A., Proud, Crystal M., Farrar, Michelle A., Kwon, Jennifer M., Saito, Kayoko, Desguerre, Isabelle, McMillan, Hugh J.
Publicado 2021

Health, wellbeing and lived experiences of adults with SMA: a scoping systematic review

por Wan, Hamish W. Y., Carey, Kate A., D’Silva, Arlene, Vucic, Steve, Kiernan, Matthew C., Kasparian, Nadine A., Farrar, Michelle A.
Publicado 2020

Dysfunction of axonal membrane conductances in adolescents and young adults with spinal muscular atrophy

por Farrar, Michelle A., Vucic, Steve, Lin, Cindy S.-Y., Park, Susanna B., Johnston, Heather M., du Sart, Desirée, Bostock, Hugh, Kiernan, Matthew C.
Publicado 2011

Emerging therapies and challenges in spinal muscular atrophy

por Farrar, Michelle A., Park, Susanna B., Vucic, Steve, Carey, Kate A., Turner, Bradley J., Gillingwater, Thomas H., Swoboda, Kathryn J., Kiernan, Matthew C.
Publicado 2017