By Professor Michelle Anne Farrar

Journal articles

MacCannell D; Berger Z; East L; Mercuri E; Kirschner J; Muntoni F; Farrar MA; Peng J; Zhou J; Nestorov I; Farwell W; Finkel RS, 2021, 'Population pharmacokinetics-based recommendations for a single delayed or missed dose of nusinersen', Neuromuscular Disorders, 31, pp. 310 - 318, http://dx.doi.org/10.1016/j.nmd.2021.02.014

Chand DH; Zaidman C; Arya K; Millner R; Farrar MA; Mackie FE; Goedeker NL; Dharnidharka VR; Dandamudi R; Reyna SP, 2021, 'Thrombotic Microangiopathy Following Onasemnogene Abeparvovec for Spinal Muscular Atrophy: A Case Series', Journal of Pediatrics, 231, pp. 265 - 268, http://dx.doi.org/10.1016/j.jpeds.2020.11.054

Kariyawasam DST; D'Silva AM; Vetsch J; Wakefield CE; Wiley V; Farrar MA, 2021, '“We needed this”: perspectives of parents and healthcare professionals involved in a pilot newborn screening program for spinal muscular atrophy', Eclinicalmedicine, 33, pp. 100742, http://dx.doi.org/10.1016/j.eclinm.2021.100742

Waddell LB; Bryen SJ; Cummings BB; Bournazos A; Evesson FJ; Joshi H; Marshall JL; Tukiainen T; Valkanas E; Weisburd B; Sadedin S; Davis MR; Faiz F; Gooding R; Sandaradura SA; O'Grady GL; Tchan MC; Mowat DR; Oates EC; Farrar MA; Sampaio H; Ma A; Neas K; Wang MX; Charlton A; Chan C; Kenwright DN; Graf N; Arbuckle S; Clarke NF; MacArthur DG; Jones KJ; Lek M; Cooper ST, 2021, 'WGS and RNA Studies Diagnose Noncoding DMD Variants in Males With High Creatine Kinase', Neurology Genetics, 7, http://dx.doi.org/10.1212/NXG.0000000000000554

Kariyawasam D; D'silva A; Howells J; Herbert K; Geelan-Small P; Lin CSY; Farrar MA, 2021, 'Motor unit changes in children with symptomatic spinal muscular atrophy treated with nusinersen', Journal of Neurology Neurosurgery and Psychiatry, 92, pp. 78 - 85, http://dx.doi.org/10.1136/jnnp-2020-324254

Shih STF; Farrar MA; Wiley V; Chambers G, 2021, 'Newborn screening for spinal muscular atrophy with disease-modifying therapies: a cost-effectiveness analysis', Journal of Neurology Neurosurgery and Psychiatry, 92, pp. 1296 - 1304, http://dx.doi.org/10.1136/jnnp-2021-326344

Strauss K; Muntoni F; Farrar M; Saito K; Mendell J; Servais L; McMillan H; Swoboda K; Kwon J; Zaidman C; Chiriboga C; Iannaccone S; Krueger J; Parsons J; Shieh P; Kavanagh S; Chand D; Tauscher-Wisniewski S; Macek T, 2021, 'Onasemnogene Abeparvovec Gene Therapy in Presymptomatic spinal muscular atrophy (SMA): SPR1NT study update in children with 3 Copies of SMN2', NEUROMUSCULAR DISORDERS, 31, pp. S133 - S133, http://dx.doi.org/10.1016/j.nmd.2021.07.299

Paguinto SG; Kasparian NA; Bray P; Farrar M, 2020, '“It’s not just the wheelchair, it’s everything else”: Australian parents’ perspectives of wheelchair prescription for children with neuromuscular disorders', Disability and Rehabilitation, 42, pp. 3457 - 3466, http://dx.doi.org/10.1080/09638288.2019.1595749

Farrar MA; Kiernan MC, 2020, 'Spinal muscular atrophy — the dawning of a new era', Nature Reviews Neurology, 16, pp. 593 - 594, http://dx.doi.org/10.1038/s41582-020-00410-7

Farrar MA; Kiernan MC, 2020, 'Treating adults with spinal muscular atrophy with nusinersen', Journal of Neurology Neurosurgery and Psychiatry, 91, pp. 1139, http://dx.doi.org/10.1136/jnnp-2020-324036

Finkel R; Castro D; Farrar M; Tulinius M; Krosschell K; Saito K; Gambino G; Foster R; Ramirez-Schrempp D; Wong J; Kandinov B; Farwell W, 2020, 'Nusinersen in infantile-onset spinal muscular atrophy: results from longer-term treatment from the open-label SHINE extension study', NEUROMUSCULAR DISORDERS, 30, pp. S124 - S124, http://dx.doi.org/10.1016/j.nmd.2020.08.265

Chou E; Lindeback R; Sampaio H; Farrar MA, 2020, 'Nutritional practices in pediatric patients with neuromuscular disorders', Nutrition Reviews, 78, pp. 857 - 865, http://dx.doi.org/10.1093/nutrit/nuz109

Kandula T; Park SB; Carey KA; Lin CSY; Farrar MA, 2020, 'Peripheral nerve maturation and excitability properties from early childhood: Comparison of motor and sensory nerves', Clinical Neurophysiology, 131, pp. 2452 - 2459, http://dx.doi.org/10.1016/j.clinph.2020.06.035

D'Silva A; Farrar MA, 2020, 'Personalized medicine for children with spinal muscular atrophy: Toward the holy grail', Muscle and Nerve, 62, pp. 425 - 426, http://dx.doi.org/10.1002/mus.27030

Farrar MA; Carey KA; Paguinto SG; Kasparian NA; De Abreu Lourenço R, 2020, '“The Whole Game is Changing and You’ve Got Hope”: Australian Perspectives on Treatment Decision Making in Spinal Muscular Atrophy', Patient, 13, pp. 389 - 400, http://dx.doi.org/10.1007/s40271-020-00415-w

Kariyawasam D; Alexander IE; Kurian M; Farrar MA, 2020, 'Great expectations: Virus-mediated gene therapy in neurological disorders', Journal of Neurology Neurosurgery and Psychiatry, 91, pp. 849 - 860, http://dx.doi.org/10.1136/jnnp-2019-322327

Chambers GM; Settumba SN; Carey KA; Cairns A; Menezes MP; Ryan M; Farrar MA, 2020, 'Prenusinersen economic and health-related quality of life burden of spinal muscular atrophy', Neurology, 95, pp. 1 - 10, http://dx.doi.org/10.1212/WNL.0000000000009715

Chiriboga CA; Darras BT; Farrar MA; Mercuri E; Kirschner J; Kuntz NL; Acsadi G; Tulinius M; Montes J; Gambino G; Foster R; Bhan I; Wong J; Kandinov B; Farwell W, 2020, 'Longer-term Treatment With Nusinersen: Results in Later-onset Spinal Muscular Atrophy From the SHINE Study (1661)', Neurology, 94, http://dx.doi.org/10.1212/wnl.94.15_supplement.1661

Castro D; Finkel RS; Farrar MA; Tulinius M; Krosschell KJ; Saito K; Gambino G; Foster R; Bhan I; Wong J; Kandinov B; Farwell W, 2020, 'Nusinersen in Infantile-onset Spinal Muscular Atrophy: Results from Longer-term Treatment from the Open-label SHINE Extension Study (1640)', Neurology, 94, http://dx.doi.org/10.1212/wnl.94.15_supplement.1640

Darras BT; De Vivo DC; Farrar MA; Mercuri E; Finkel RS; Foster R; Farwell W; Bhan I, 2020, 'Safety Profile of Nusinersen in Presymptomatic and Infantile-Onset Spinal Muscular Atrophy (SMA): Interim Results From the NURTURE and ENDEAR-SHINE Studies (1659)', Neurology, 94, http://dx.doi.org/10.1212/wnl.94.15_supplement.1659

Kandula T; Farrar MA; Cohn RJ; Carey KA; Johnston K; Kiernan MC; Krishnan AV; Park SB, 2020, 'Changes in long term peripheral nerve biophysical properties in childhood cancer survivors following neurotoxic chemotherapy', Clinical Neurophysiology, 131, pp. 783 - 790, http://dx.doi.org/10.1016/j.clinph.2019.12.411

Wan HWY; Carey KA; D'Silva A; Vucic S; Kiernan MC; Kasparian NA; Farrar MA, 2020, 'Health, wellbeing and lived experiences of adults with SMA: A scoping systematic review', Orphanet Journal of Rare Diseases, 15, pp. 70, http://dx.doi.org/10.1186/s13023-020-1339-3

Kariyawasam DST; Russell JS; Wiley V; Alexander IE; Farrar MA, 2020, 'The implementation of newborn screening for spinal muscular atrophy: the Australian experience', Genetics in Medicine, 22, pp. 557 - 565, http://dx.doi.org/10.1038/s41436-019-0673-0

Stelzer-Braid S; Wynn M; Chatoor R; Scotch M; Ramachandran V; Teoh HL; Farrar MA; Sampaio H; Andrews PI; Craig ME; MacIntyre CR; Varadhan H; Kesson A; Britton PN; Newcombe J; Rawlinson WD, 2020, 'Next generation sequencing of human enterovirus strains from an outbreak of enterovirus A71 shows applicability to outbreak investigations', Journal of Clinical Virology, 122, http://dx.doi.org/10.1016/j.jcv.2019.104216

Kariyawasam DST; D’Silva A; Herbert K; Lin CSY; Geelan-Small P; Farrar MA, 2020, '7. Motor unit changes in children with spinal muscular atrophy treated with nusinersen', Clinical Neurophysiology, 131, pp. e3 - e4, http://dx.doi.org/10.1016/j.clinph.2019.11.040

Mercuri E; Darras B; Chiriboga C; Farrar M; Kirschner J; Kuntz N; Acsadi G; Tulinius M; Montes J; Gambino G; Foster R; Ramirez-Schrempp D; Garafolo S; Farwell W, 2020, 'Longer-term treatment with nusinersen: Results in later-onset spinal muscular atrophy from the SHINE study', NEUROMUSCULAR DISORDERS, 30, pp. S121 - S121, http://dx.doi.org/10.1016/j.nmd.2020.08.256

Strauss K; Farrar M; Swoboda K; Saito K; Chiriboga C; Finkel R; Iannaccone S; Krueger J; Kwon J; McMillan H; Servais L; Mendell J; Parsons J; Scoto M; Shieh P; Zaidman C; Schultz M; Ogrinc F; Group S; Muntoni F, 2020, 'Onasemnogene abeparvovec gene therapy in presymptomatic spinal muscular atrophy (SMA): SPR1NT study update', NEUROMUSCULAR DISORDERS, 30, pp. S122 - S122, http://dx.doi.org/10.1016/j.nmd.2020.08.259

Jones HF; Bryen SJ; Waddell LB; Bournazos A; Davis M; Farrar MA; McLean CA; Mowat DR; Sampaio H; Woodcock IR; Ryan MM; Jones KJ; Cooper ST, 2019, 'Importance of muscle biopsy to establish pathogenicity of DMD missense and splice variants', Neuromuscular Disorders, 29, pp. 913 - 919, http://dx.doi.org/10.1016/j.nmd.2019.09.013

Donkervoort S; Sabouny R; Yun P; Gauquelin L; Chao KR; Hu Y; Al Khatib I; Töpf A; Mohassel P; Cummings BB; Kaur R; Saade D; Moore SA; Waddell LB; Farrar MA; Goodrich JK; Uapinyoying P; Chan SHS; Javed A; Leach ME; Karachunski P; Dalton J; Medne L; Harper A; Thompson C; Thiffault I; Specht S; Lamont RE; Saunders C; Racher H; Bernier FP; Mowat D; Witting N; Vissing J; Hanson R; Coffman KA; Hainlen M; Parboosingh JS; Carnevale A; Yoon G; Schnur RE; Boycott KM; Mah JK; Straub V; Foley AR; Innes AM; Bönnemann CG; Shutt TE, 2019, 'MSTO1 mutations cause mtDNA depletion, manifesting as muscular dystrophy with cerebellar involvement', Acta Neuropathologica, 138, pp. 1013 - 1031, http://dx.doi.org/10.1007/s00401-019-02059-z

Paguinto SG; Kasparian NA; Bray P; Farrar M, 2019, 'Parents’ perceptions of power wheelchair prescription for children with a neuromuscular disorder: a scoping review', Disability and Rehabilitation, 41, pp. 2750 - 2757, http://dx.doi.org/10.1080/09638288.2018.1474496

Strauss KA; Swoboda KJ; Farrar MA; McMillan HJ; Parsons J; Krueger JM; Iannaccone ST; Chiriboga CA; Kwon JM; Saito K; Scoto M; Baldinetti F; Schultz M; Kernbauer E; Farrow M; Ogrinc FG; Kavanagh S; Feltner DE; McGill BE; Spector SA; L'Italien J; Sproule DM; Muntoni F, 2019, 'Onasemnogene abeparvovec gene-replacement therapy (GRT) in presymptomatic spinal muscular atrophy (SMA): SPR1NT study update', JOURNAL OF THE NEUROLOGICAL SCIENCES, 405, http://dx.doi.org/10.1016/j.jns.2019.10.1317

Darras BT; Farrar MA; Mercuri E; Finkel RS; Foster R; Hughes SG; Bhan I; Farwell W; Gheuens S, 2019, 'An Integrated Safety Analysis of Infants and Children with Symptomatic Spinal Muscular Atrophy (SMA) Treated with Nusinersen in Seven Clinical Trials', CNS Drugs, 33, pp. 919 - 932, http://dx.doi.org/10.1007/s40263-019-00656-w

Schultz M; Swoboda K; Farrar M; McMillan H; Parsons J; Wells C; Ogrinc FG; McGill B; Feltner DE; L’Italien J; Sproule DM; Strauss KA, 2019, 'AVXS-101 Gene-Replacement Therapy (GRT) in Presymptomatic Spinal Muscular Atrophy (SMA): Study Update (P1.6-057)', Neurology, 92, http://dx.doi.org/10.1212/wnl.92.15_supplement.p1.6-057

Finkel RS; Castro D; Farrar MA; Tulinius M; Krosschell KJ; Saito K; Zhang Y; Bhan I; Farwell W; Reyna SP, 2019, 'Interim Report on the Safety and Efficacy of Longer-Term Treatment With Nusinersen in Infantile-Onset Spinal Muscular Atrophy (SMA): Updated Results From the SHINE Study (S25.004)', Neurology, 92, http://dx.doi.org/10.1212/wnl.92.15_supplement.s25.004

Darras BT; Farrar MA; Mercuri E; Chiriboga CA; Kirschner J; Kuntz N; Shieh P; Tulinius M; Montes J; Gambino G; Foster R; Bhan I; Farwell W; Reyna SP, 2019, 'Interim Report on the Safety and Efficacy of Longer-term Treatment With Nusinersen in Later-onset Spinal Muscular Atrophy (SMA): Results From the SHINE Study (P1.6-063)', Neurology, 92, http://dx.doi.org/10.1212/wnl.92.15_supplement.p1.6-063

Wan HWY; Carey KA; D'Silva A; Kasparian NA; Farrar MA, 2019, '"Getting ready for the adult world": How adults with spinal muscular atrophy perceive and experience healthcare, transition and well-being', Orphanet Journal of Rare Diseases, 14, pp. 74, http://dx.doi.org/10.1186/s13023-019-1052-2

Mandarakas MR; Menezes MP; Rose KJ; Shy R; Eichinger K; Foscan M; Estilow T; Kennedy R; Herbert K; Bray P; Refshauge K; Ryan MM; Yiu EM; Farrar M; Sampaio H; Moroni I; Pagliano E; Pareyson D; Yum SW; Herrmann DN; Acsadi G; Shy ME; Burns J; Sanmaneechai O, 2019, 'Erratum: Development and validation of the charcot-marie-tooth disease infant scale (Brain (2018) 141 (3319-3330) DOI: 10.1093/brain/awy280)', Brain, 142, pp. E14, http://dx.doi.org/10.1093/brain/awy332

Cornett KMD; Wojciechowski E; Sman AD; Walker T; Menezes MP; Bray P; Halaki M; Burns J; Sman D; Cornett KMD; Mandarakas MR; Rose KJ; Bray P; Baldwin J; McKay MJ; Refshauge KM; Raymond J; Sampaio H; Mudge A; Purcell L; Miller C; Gray K; Harman M; Gabrael N; Ouvrier RA; Farrar M, 2019, 'Magnetic resonance imaging of the anterior compartment of the lower leg is a biomarker for weakness, disability, and impaired gait in childhood Charcot–Marie–Tooth disease', Muscle and Nerve, 59, pp. 213 - 217, http://dx.doi.org/10.1002/mus.26352

Kariyawasam DST; D’silva A; Lin C; Ryan MM; Farrar MA; D'Silva A, 2019, 'Biomarkers and the development of a personalized medicine approach in spinal muscular atrophy', Frontiers in Neurology, 10, pp. 898, http://dx.doi.org/10.3389/fneur.2019.00898

Day JW; Chiriboga CA; Crawford TO; Darras BT; Finkel RS; Connolly AM; Iannaccone ST; Kuntz NL; Pena LDM; Schultz M; Shieh PB; Smith EC; Farrar M; Feltner DE; Ogrinc FG; Macek TA; Kernbauer E; Muehring LM; L'Italien J; Sproule DM; Kaspar BK; Mendell JR, 2019, 'Avxs-101 gene-replacement therapy (GRT) for spinal muscular atrophy type 1 (SMA1): pivotal phase 3 study (STR1VE) update', JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY, 90, http://dx.doi.org/10.1136/jnnp-2019-anzan.58

Farrar M; Swoboda KJ; Schultz M; McMillan H; Parsons J; Alexander IE; Kernbauer E; Farrow M; Ogrinc FG; Feltner DE; McGill BE; Spector SA; L'Italien J; Sproule DM; Strauss KA, 2019, 'AVXS-101 GENE-REPLACEMENT THERAPY (GRT) IN PRESYMPTOMATIC SPINAL MUSCULAR ATROPHY (SMA): STUDY UPDATE', JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY, 90, http://dx.doi.org/10.1136/jnnp-2019-anzan.14

Strauss KA; Swoboda KJ; Farrar MA; McMillan HJ; Parsons J; Krueger JM; Iannaccone ST; Chiriboga CA; Kwon JM; Saito K; Scoto M; Kausar I; Schultz M; Kernbauer E; Farrow M; Ogrinc FG; Kavanagh S; Feltner DE; McGill BE; Spector SA; L'Italien J; Sproule DM; Muntoni F, 2019, 'AVXS-101 IN PRESYMPTOMATIC SPINAL MUSCULAR ATROPHY (SMA)', JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY, 90, pp. E14 - E14, http://dx.doi.org/10.1136/jnnp-2019-ABN-2.20

Banuelos R; Beavis E; Le Marne F; Rowe N; Gao Y; Menzies J; Farrar MA; Cardamone M, 2019, 'Factors Affecting the Efficacy, Tolerability and Compliance of Dietary Therapy for Epilepsy- Four-Years Experience', Neurological Case Reports, 2, pp. 1010 - 1010, http://www.remedypublications.com/neurological-case-reports-abstract.php?aid=4936

Schultz M; Swoboda K; Farrar M; McMillan H; Parsons J; Kernbauer E; Farrow M; Ogrinc F; Kavanagh S; Feltner D; McGill B; Spector S; L'Italien J; Sproule D; Strauss K, 2019, 'Onasemnogene abeparvovec gene-replacement therapy (GRT) in pre-symptomatic spinal muscular atrophy (SMA)', NEUROMUSCULAR DISORDERS, 29, pp. S183 - S183, http://dx.doi.org/10.1016/j.nmd.2019.06.512

Schultz M; Swoboda K; Farrar M; McMillan H; Parsons J; Farrow M; Ogrinc FG; Feltner DE; McGill BE; Spector SA; L’Italien J; Sproule DM; Strauss KA, 2019, 'P.065 AVXS-101 gene-replacement therapy (GRT)) in presymptomatic spinal muscular atrophy (SMA): study update', Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques, 46, pp. S31 - S32, http://dx.doi.org/10.1017/cjn.2019.165

Mandarakas MR; Menezes MP; Rose KJ; Shy R; Eichinger K; Foscan M; Estilow T; Kennedy R; Herbert K; Bray P; Refshauge K; Ryan MM; Yiu EM; Farrar M; Sampaio H; Moroni I; Pagliano E; Pareyson D; Yum SW; Herrmann DN; Acsadi G; Shy ME; Burns J; Sanmaneechai O, 2018, 'Development and validation of the Charcot-Marie-Tooth disease infant scale', Brain, 141, pp. 3319 - 3330, http://dx.doi.org/10.1093/brain/awy280

Castro D; Farrar M; Finkel R; Tulinius M; Krosschell K; Saito K; Zhang Y; Bhan I; Farwell W; Reyna S, 2018, 'Interim report on the safety and efficacy of longer-term treatment with nusinersen in infantile-onset spinal muscular atrophy: results from the SHINE study', NEUROMUSCULAR DISORDERS, 28, pp. S79 - S80, http://dx.doi.org/10.1016/j.nmd.2018.06.198

Chen KA; Farrar M; Cardamone M; Gill D; Smith R; Cowell CT; Truong L; Lawson JA, 2018, 'Cannabidiol for treating drug-resistant epilepsy in children: The New South Wales experience', Medical Journal of Australia, 209, pp. 217 - 221, http://dx.doi.org/10.5694/mja18.00023

Fitzgerald DA; Abel F; Jones KJ; Farrar MA, 2018, 'Spinal muscular atrophy: A modifiable disease emerges', Paediatric Respiratory Reviews, 28, pp. 1 - 2, http://dx.doi.org/10.1016/j.prrv.2018.07.001

Sampaio H; Wilcken B; Farrar M, 2018, 'Screening for spinal muscular atrophy', The Medical journal of Australia, 209, pp. 147 - 148, http://dx.doi.org/10.5694/mja17.00772

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