By Professor Michelle Anne Farrar

Conference Abstracts

Darras B; Hagenacker T; Finkel R; Mercuri E; Montes J; Kuntz N; Farrar M; Sansone V; Berger Z; MacCannell D; Shen C; Paradis A; Bohn J; Wagner J; Somera-Molina K, 2022, 'Rationale/design of the phase 3b ASCEND study of investigational higher dose nusinersen in participants with SMA previously treated with risdiplam', in NEUROMUSCULAR DISORDERS, PERGAMON-ELSEVIER SCIENCE LTD, Vol. 32, pp. S86 - S86, http://dx.doi.org/10.1016/j.nmd.2022.07.185

Strauss K; Muntoni F; Farrar M; Saito K; Mendell J; Servals L; McMillan H; Finkel R; Swoboda K; Kwon J; Zaidman C; Chiriboga C; Iannaccone S; Krueger J; Parsons J; Shieh P; Kavanagh S; Chand D; Tauscher-Wisniewski S; McGill B; Macek T, 2022, 'Onasemnogene Abeparvovec for Presymptomatic Infants with Spinal Muscular Atrophy and Two Copies of SMN2', in NEUROLOGY, LIPPINCOTT WILLIAMS & WILKINS, WA, Seattle, Vol. 98, presented at Annual Meeting of the American-Academy-of-Neurology, WA, Seattle, 02 April 2022 - 07 April 2022, https://www.webofscience.com/api/gateway?GWVersion=2&SrcApp=PARTNER_APP&SrcAuth=LinksAMR&KeyUT=WOS:000894020500383&DestLinkType=FullRecord&DestApp=ALL_WOS&UsrCustomerID=891bb5ab6ba270e68a29b250adbe88d1

Strauss K; Muntoni F; Farrar M; Saito K; Mendell J; Servais L; McMillan H; Finkel R; Swoboda K; Kwon J; Zaidman C; Chiriboga C; Lannaccone S; Krueger J; Parsons J; Shieh P; Kavanagh S; Chand D; Tauscher-Wisniewski S; McGill B; Macek T, 2022, 'Onasemnogene Abeparvovec in Presymptomatic Spinal Muscular Atrophy (SMA): SPRINT Study Update in Children with Three Copies of SMN2', in NEUROLOGY, LIPPINCOTT WILLIAMS & WILKINS, WA, Seattle, Vol. 98, presented at Annual Meeting of the American-Academy-of-Neurology, WA, Seattle, 02 April 2022 - 07 April 2022, https://www.webofscience.com/api/gateway?GWVersion=2&SrcApp=PARTNER_APP&SrcAuth=LinksAMR&KeyUT=WOS:000894020500058&DestLinkType=FullRecord&DestApp=ALL_WOS&UsrCustomerID=891bb5ab6ba270e68a29b250adbe88d1

Finkel RS; Farrar MA; Vlodavets D; Servais L; Zanoteli E; Al-Muhaizea M; Nelson L; Prufer A; Wang Y; Fisher C; Gerber M; Gorni K; Kletzl H; Palfreeman L; Scalco R; Bertini E, 2022, 'RAINBOWFISH: Preliminary Efficacy and Safety Data in Risdiplam-Treated Infants with Presymptomatic SMA', in NEUROLOGY, LIPPINCOTT WILLIAMS & WILKINS, WA, Seattle, Vol. 98, presented at Annual Meeting of the American-Academy-of-Neurology, WA, Seattle, 02 April 2022 - 07 April 2022, https://www.webofscience.com/api/gateway?GWVersion=2&SrcApp=PARTNER_APP&SrcAuth=LinksAMR&KeyUT=WOS:000894020500235&DestLinkType=FullRecord&DestApp=ALL_WOS&UsrCustomerID=891bb5ab6ba270e68a29b250adbe88d1

Gonski K; Chuang S; Teng A; Thambipillay G; Farrar M; Menezes M; Fitzgerald D, 2022, 'Respiratory outcomes before and after the introduction of nusinersen in patients with spinal muscular atrophy (Types 1-3)', in RESPIROLOGY, WILEY, Vol. 27, pp. 63 - 63, https://www.webofscience.com/api/gateway?GWVersion=2&SrcApp=PARTNER_APP&SrcAuth=LinksAMR&KeyUT=WOS:000769654800098&DestLinkType=FullRecord&DestApp=ALL_WOS&UsrCustomerID=891bb5ab6ba270e68a29b250adbe88d1

Crawford T; Swoboda K; Sumner C; Farrar M; Muntoni F; Finkel R; Mercuri E; Sohn J; Petrillo M; Chin R; Jiang X; Paradis A, 2021, 'Phosphorylated neurofilament heavy chain level is associated with future motor function in nusinersen-treated individuals with infantile-onset SMA', in NEUROMUSCULAR DISORDERS, PERGAMON-ELSEVIER SCIENCE LTD, ELECTR NETWORK, Vol. 31, pp. S137 - S137, presented at Virtual Congress of the World-Muscle-Society, ELECTR NETWORK, 20 September 2021 - 24 September 2021, http://dx.doi.org/10.1016/j.nmd.2021.07.311

Servais L; Al-Muhaizea M; Farrar M; Nelson L; Prufer A; Finkel R; Wang Y; Zanoteli E; El-Khairi M; Gerber M; Gorni K; Kletzl H; Palfreeman L; Scalco R; Bertini E, 2021, 'RAINBOWFISH: A study of risdiplam in infants with presymptomatic spinal muscular atrophy (SMA)', in NEUROMUSCULAR DISORDERS, PERGAMON-ELSEVIER SCIENCE LTD, ELECTR NETWORK, Vol. 31, pp. S48 - S48, presented at Virtual Congress of the World-Muscle-Society, ELECTR NETWORK, 20 September 2021 - 24 September 2021, http://dx.doi.org/10.1016/j.nmd.2021.07.021

Bertini E; Al-Muhaizea M; Farrar M; Nelson L; Araujo APDQC; Servais L; Wang Y; Zanoteli E; El-Khairi M; Gerber M; Gorni K; Kletzl H; Palfreeman L; Scalco RS; Finkel R, 2021, 'RAINBOWFISH: A study of risdiplam in infants with presymptomatic SMA', in EUROPEAN JOURNAL OF NEUROLOGY, WILEY, Vol. 28, pp. 396 - 396, https://www.webofscience.com/api/gateway?GWVersion=2&SrcApp=PARTNER_APP&SrcAuth=LinksAMR&KeyUT=WOS:000663065900583&DestLinkType=FullRecord&DestApp=ALL_WOS&UsrCustomerID=891bb5ab6ba270e68a29b250adbe88d1

Frohlich M; Widger J; Thambipillay G; Teng A; Farrar M; Chuang S, 2021, 'PREDICTORS OF NOCTURNAL HYPOVENTILATION IN PAEDIATRIC NEUROMUSCULAR PATIENTS: FVC Z-SCORE', in RESPIROLOGY, WILEY, Vol. 26, pp. 190 - 190, https://www.webofscience.com/api/gateway?GWVersion=2&SrcApp=PARTNER_APP&SrcAuth=LinksAMR&KeyUT=WOS:000644657800360&DestLinkType=FullRecord&DestApp=ALL_WOS&UsrCustomerID=891bb5ab6ba270e68a29b250adbe88d1

Frohlich M; Widger J; Thambipillay G; Teng A; Farrar M; Chuang S, 2020, 'Daytime predictors of nocturnal hypercapnic hypoventilation in children with neuromuscular disorders: a threshold for FVC z-score', in EUROPEAN RESPIRATORY JOURNAL, EUROPEAN RESPIRATORY SOC JOURNALS LTD, Vol. 56, http://dx.doi.org/10.1183/13993003.congress-2020.1223

Mueller-Felber W; Darras BT; Chiriboga CA; Farrar MA; Mercuri E; Kirschner J; Kuntz NL; Acsadi G; Tulinius M; Montes J; Gambino G; Foster R; Ramirez-Schrempp D; Wong J; Kandinov B; Farwell W, 2020, 'Longer-term Nusinersen Treatment According to Age at First Dose: Results From the SHINE Study in Later-onset Spinal Muscular Atrophy', in EUROPEAN JOURNAL OF NEUROLOGY, WILEY, ELECTR NETWORK, Vol. 27, pp. 221 - 221, presented at 6th Congress of the European-Academy-of-Neurology (EAN), ELECTR NETWORK, 23 May 2020 - 26 May 2020, https://www.webofscience.com/api/gateway?GWVersion=2&SrcApp=PARTNER_APP&SrcAuth=LinksAMR&KeyUT=WOS:000534616800321&DestLinkType=FullRecord&DestApp=ALL_WOS&UsrCustomerID=891bb5ab6ba270e68a29b250adbe88d1

Kirschner J; Darras B; Farrar M; Mercuri E; Chiriboga C; Kuntz N; Shieh P; Tulinius M; Montes J; Reyna S; Gambino G; Foster R; Bhan I; Wong J; Farwell W, 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', in Neuromuscular Disorders, Elsevier, DENMARK, Copenhagen, Vol. 29, pp. s184, presented at 24th International Annual Congress of the World-Muscle-Society (WMS), DENMARK, Copenhagen, 01 October 2019 - 05 October 2019, http://dx.doi.org/10.1016/j.nmd.2019.06.514

Kariyawasam DS; Samapio H; Mowat D; Farrar M, 2019, 'GENETIC CARRIER SCREENING FOR DUCHENNE MUSCULAR DYSTROPHY: THE OUTCOME OF OVER TWENTY YEARS OF GENETIC COUNSELLING ON DISEASE EPIDEMIOLOGY IN A SINGLE-CENTRE COHORT STUDY IN NEW SOUTH WALES (NSW), AUSTRALIA', in JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY, BMJ PUBLISHING GROUP, AUSTRALIA, Sydney, Vol. 90, presented at Annual Scientific Meeting of the Australian-and-New Zealand-Association-of-Neurologists (ANZAN), AUSTRALIA, Sydney, 21 May 2019 - 24 May 2019, http://dx.doi.org/10.1136/jnnp-2019-anzan.22

Seow C; Farrar M; Doumit M; Thambipillay G; Widger J; Jaffe A; Chuang SY, 2019, 'Compliance to Respiratory Care Guidelines for Children with Neuromuscular Disease at Sydney Children's Hospital', in AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE, AMER THORACIC SOC, TX, Dallas, Vol. 199, presented at International Conference of the American-Thoracic-Society, TX, Dallas, 17 May 2019 - 22 May 2019, https://www.webofscience.com/api/gateway?GWVersion=2&SrcApp=PARTNER_APP&SrcAuth=LinksAMR&KeyUT=WOS:000466771104303&DestLinkType=FullRecord&DestApp=ALL_WOS&UsrCustomerID=891bb5ab6ba270e68a29b250adbe88d1

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', in NEUROLOGY, LIPPINCOTT WILLIAMS & WILKINS, PA, Philadelphia, Vol. 92, presented at 71st Annual Meeting of the American-Academy-of-Neurology (AAN), PA, Philadelphia, 04 May 2019 - 10 May 2019, https://www.webofscience.com/api/gateway?GWVersion=2&SrcApp=PARTNER_APP&SrcAuth=LinksAMR&KeyUT=WOS:000475965906183&DestLinkType=FullRecord&DestApp=ALL_WOS&UsrCustomerID=891bb5ab6ba270e68a29b250adbe88d1

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', in NEUROLOGY, LIPPINCOTT WILLIAMS & WILKINS, PA, Philadelphia, Vol. 92, presented at 71st Annual Meeting of the American-Academy-of-Neurology (AAN), PA, Philadelphia, 04 May 2019 - 10 May 2019, https://www.webofscience.com/api/gateway?GWVersion=2&SrcApp=PARTNER_APP&SrcAuth=LinksAMR&KeyUT=WOS:000475965900302&DestLinkType=FullRecord&DestApp=ALL_WOS&UsrCustomerID=891bb5ab6ba270e68a29b250adbe88d1

Farrar MA; Tomlinson SE; Howells J; Lin CS-Y; Carey K; Park SB; Hollingsworth G; Lawson JA; Kiernan MC; Berkovic SF; Burke D; Scheffer IE, 2019, '009 Axonal excitability properties in dravet’s syndrome reflect effect of loss of sodium channels', in Journal of Neurology, Neurosurgery & Psychiatry, BMJ, Vol. 90, pp. A4.1 - A4, http://dx.doi.org/10.1136/jnnp-2019-anzan.9

Farrar MA; KAndula T; Murray J; Goldstein D; Krishnan A; PArk S, 2016, 'Subclinical peripheral neuropathy prior to chemotherapy in colorectal cancer patients – Myth or reality?', in Subclinical peripheral neuropathy prior to chemotherapy in colorectal cancer patients – Myth or reality?, Elsevier, Vol. 127, pp. e13, presented at ANZAN Biennial Clinical Neurophysiology Workshop, http://dx.doi.org/10.1016/j.clinph.2015.11.029

Farrar MA; Chew K; Widger J, 2015, 'The Impact Of Nutrition On Respiratory Function In Duchenne Muscular Dystrophy', in The Impact Of Nutrition On Respiratory Function In Duchenne Muscular Dystrophy, presented at Australian Thoracic Society ASM

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