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Michelle A. Farrar
Researcher at Boston Children's Hospital
Publications - 127
Citations - 3874
Michelle A. Farrar is an academic researcher from Boston Children's Hospital. The author has contributed to research in topics: Medicine & Spinal muscular atrophy. The author has an hindex of 23, co-authored 96 publications receiving 2473 citations. Previous affiliations of Michelle A. Farrar include University of New South Wales & University of Sydney.
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Journal ArticleDOI
Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy
Richard S. Finkel,Eugenio Mercuri,Basil T. Darras,Anne M. Connolly,Nancy L. Kuntz,Janbernd Kirschner,Claudia A. Chiriboga,Kayoko Saito,Laurent Servais,Eduardo F. Tizzano,Haluk Topaloglu,Mar Tulinius,Jacqueline Montes,Allan M. Glanzman,Kathie M. Bishop,Z. John Zhong,Sarah Gheuens,C. Frank Bennett,Eugene Schneider,Wildon Farwell,Darryl C. De Vivo,Walter G. Bradley,M. K. Schroth,J. B. Bodensteriner,C. S. Davis,R. Shell,J. Hen,E. D. Austin,S. Aziz-Zaman,J. Cappell,Andrei Constantinescu,Rosangel Cruz,Jahannaz Dastgir,Sally Dunaway,K. Engelstad,M. Gormley,N. Holuba La Marca,Alexander G. Khandji,S. Kramer,Jonathan Marra,C. Ortiz-Miller,M. Popolizio,Rachel Salazar,L. Sanabria,L. Weimer,Pallavi Anand,R. Gadeken,Paul T. Golumbek,Catherine Siener,Craig M. Zaidman,Fouad Al-Ghamdi,C. Berde,Partha S. Ghosh,Robert J. Graham,Tim Harrington,A. Koka,R. Laine,W. Liew,Elizabeth Mirek,G. Ordonez,Amy Pasternak,Janet Quigley,Navil F. Sethna,M. Souris,Heather Szelag,L. Wand,John W. Day,Genevieve D'Souza,T. T. Duong,Richard Gee,J. Kitsuwa-Lowe,D. McFall,S. Patnaik,S. Paulose,Jennifer Perez,Christopher Proud,B. Purse,R. J. Ramamurthi,Sarada Sakamuri,Jacinda B. Sampson,B. Sanjanwala,A. C. Tesi Rocha,K. Watson,L. Welsh,L.D.M. Peña,Laura E. Case,J. Coates,Stephanie DeArmey,M. M. Homi,C. Milleson,N. Nelson,Alexandra C. Ross,Edward C. Smith,B. Taicher,J. Wootton,Erika Finanger,D. Benjamin,A. Frank,Catherine E. Roberts,Barry S. Russman,K. Zilke,D. Berry,Matthew Civitello,D. Cook,J. D. Endsley,C. Johnson,M. Kasper,W. Leon,A. Lim,K. O'Reardon,L. Y. Sigurdardottir,J. Turner,F. Weber-Guzman,M. Zinn,Susan T. Iannaccone,Diana Castro,M. Cowie,Alan C Farrow-Gillespie,A. Herbert,M. Kauk,D. McElroy,N. Miller,Leslie Nelson,Luke Smith,T. Spain,S. Trest,Nicholas E. Johnson,Russell J. Butterfield,D. DiBella,K. Mayne,T. M. Newcomb,N. Rausch,C. Blomgren,H. W. Choi,Leon G. Epstein,S. Goldman,Kristin J. Krosschell,Jena M. Krueger,Jonathan E. Kurz,Vamshi K. Rao,Julie A. Parsons,V. Allen,Alan Bielsky,K. Booker,A. Camuto,Terri Carry,Peter G. Fuhr,Melissa Gibbons,J. Janas,H. Johnson,C. Kelly,L. S. Lord-Halvorson,S. Nicolarsen,S. Shea,V. Tran,G. VanderVeen,M. Yang,C. Zimmerman,Perry B. Shieh,N. Parziale,L. Rao,J. W. Said,F. Shu,C. Skura,L. Staudt,Gihan Tennekoon,L. Adang,John F. Brandsema,M. Chadehumbe,Jean Flickinger,Elizabeth Kichula,D. Stanford,M. Toms,J. Zigmont,Maryam Oskoui,S. Arpin,P. Dinunzio,P. M. Ingelmo,C. Poulin,G. Rivera,C. Sabapathy,M. Srour,S. Turgeon-Desilet,D. Zielinski,Kathryn Selby,C. King,J. Lee,A. Michoulas,E. Roland,Jiri Vajsar,V. Chau,James J. Dowling,R. Haldenby,M. Miki,S. So,S. I. Pascual Pascual,A. Martinez Bermejo,S. Epinosa Garcia,S. Garcia Guixot,M. M. Martinez Moreno,M. del Pilar Tirado Requero,M. del Mar Garcia Romero,C. Aguilar,F. Munell Casadesus,M. B. Gomez Garcia de la Banda,M. Gallardo,G. Gili,M. Alavarez Molinero,M. de Los Angeles Tormos Munoz,N. J. Palacios,B. Planas Pascual,M. del Mar Melendez Plumed,A. F. Rucian,E. Toro Tamargo,M. Gratacos Vinola,S. Borell,Matthias Eckenweiler,Marcus Krüger,Astrid Pechmann,B. Rippberger,S. Stein,Sibylle Vogt,S. Wider,Ulrike Schara,B. Andres,A. Della Marina,A. Ganfuss,P. Jachertz,Heike Koelbel,K. Rupprich,E. S. Schroers,N. Sponemann,Claudio Bruno,Chiara Fiorillo,Alberto Garaventa,Paola Lanteri,Valentina Lanzillotta,C. Manzitti,M. Pedermonte,Paola Tacchetti,Federica Trucco,A. Zuffi,R. De Sanctis,Lavinia Fanelli,Marco Luigetti,Concetta Palermo,Marika Pane,Marco Piastra,Serena Sivo,E. Gargaun,Teresa Gidaro,S. Gilabert,P. L. Léger,A. G. Le Moing,C. Lilien,Michèle Mayer,Q. Ollievier,J. Rambaud,Jessica Taytard,R. Vialle,T. Voit,Francesco Muntoni,L. D'Argenzio,PH Lister,Adnan Y. Manzur,J. Pisco Domingos,Danielle Ramsey,Valeria Ricotti,L. Schottlaender,Mariacristina Scoto,S. M. Scuplak,V. Selby,Volker Straub,S. Baily,Marta Bertoli,A. Mayhew,Robert Muni Lofra,Adrian Murphy,Claire L Wood,Niklas Darin,J. Eldblom,E. Kimber,A. K. Kroksmark,A. Lindstedt,E. Michael,Kalliopi Sofou,Nicolas Deconinck,A. Christiaens,Sandra Coppens,K. DeCock,E. De Vos voor,F. Dorban,G. Gilbert,S. Rooze,V. Tahon,R. Van Coster,R. Van Der Looven,Arnaud Vanlander,D. Vens,Helene Verhelst,B. Wenderickx,S. Wittevrongel,Michelle A. Farrar,N. Berthon-Jones,Michael Doumit,Karen Herbert,Tejaswi Kandula,Margot Morrison,J. O'Brien,S. Richardson,H. A. Ferreira Sampaio,H. L. Teoh,Monique M. Ryan,K. Carroll,K. L. De Valle,D. Villano,Ian R. Woodcock,E. M. Yiu,Didem Ardicli,C. Gunbey,V. G. Haliloglu,Ayşe Karaduman,Bahadır Konuşkan,F. G. Yildiz Sarikaya,E. Serdaroglu,M. Tanyildiz,C. M. Temucin,Mirac Yildirim,Öznur Yilmaz,R. Arakawa,Y. Chiba,K. Eto,K. Hirasawa,T. Ikai,S. Ito,Y. Ito,Y. Kaburagi,H. Kaneko,S. Matsumaru,N. Matsushima,K. Mizuochi,S. Nagata,H. Nakatsukasa,A. Nishikawa,Y. Otani,T. Sato,M. Shichiji,K. Sugimoto,A. Takeshita,T. Yanagishita,A. Yamauchi,Y. Takeshima,T. Fujino,N. Fukuda,T. Lee,K. Oriyama,T. Shibano,H. Shimomura,T. Tachikawa,Y. Tanaka,N. Taniguchi,Jong-Hee Chae,S. A. Choi,S. M. Chun,H. Jo,H. Kim,S. Y. Kim,J. S. Lee,B. C. Lim,Hyung Ik Shin,W. S. Son,Sophelia H. S. Chan,A. C. Chung,C. S. Yan,C. Stella,C. K.W. Joseph,C. S. Ng,H. C.C. Alvin,I. J.K. Janice,L. W.M. Wendy,M. N. Chui-San,N. Y. Ki,T. N. Shun,W. Y. Connie,W. C. Virginia,Y. Yvonne,Y. J. Jong,Tai-Heng Chen,P. C. Chou,Y. H. Chou,H. W. Chung,J. H. Hsu,Y. H. Ju,W. C. Liang,H. H. Shih,H. Y. Wang,Y. C. Wu,Y. S. Zeng +397 more
TL;DR: Those who received nusinersen were more likely to be alive and have improvements in motor function than those in the control group and infants with a shorter disease duration at screening wereMore likely than those with a longer disease duration to benefit from nusineren.
Journal ArticleDOI
Emerging therapies and challenges in Spinal Muscular Atrophy
Michelle A. Farrar,Susanna B. Park,Steve Vucic,Kate A. Carey,Bradley J. Turner,Thomas H. Gillingwater,Kathryn J. Swoboda,Matthew C. Kiernan +7 more
TL;DR: Improved understanding of the molecular basis of SMA, disease pathogenesis, natural history, and recognition of the impact of standardized care on outcomes has yielded progress toward the development of novel therapeutic strategies, which are summarized.
Journal ArticleDOI
Identification of KLHL41 Mutations Implicates BTB-Kelch-Mediated Ubiquitination as an Alternate Pathway to Myofibrillar Disruption in Nemaline Myopathy
Vandana Gupta,Gianina Ravenscroft,Ranad Shaheen,Emily J. Todd,Lindsay C. Swanson,Masaaki Shiina,Kazuhiro Ogata,Cynthia P. Hsu,Nigel F. Clarke,Basil T. Darras,Michelle A. Farrar,Amal M. Hashem,Nicholas Manton,Francesco Muntoni,Kathryn N. North,Sarah A. Sandaradura,Ichizo Nishino,Yukiko K. Hayashi,Caroline Sewry,Elizabeth Thompson,Elizabeth Thompson,Kyle S. Yau,Catherine A. Brownstein,Timothy W. Yu,Richard J.N. Allcock,Mark R. Davis,Carina Wallgren-Pettersson,Naomichi Matsumoto,Fowzan S. Alkuraya,Nigel G. Laing,Alan H. Beggs +30 more
TL;DR: These studies expand the genetic heterogeneity of NM and implicate a critical role of BTB-Kelch family members in maintenance of sarcomeric integrity in NM.
Journal ArticleDOI
The Genetics of Spinal Muscular Atrophy: Progress and Challenges
TL;DR: This review incorporates an overview of the clinical manifestations and genetics of SMA, and describes recent advances in the understanding of mechanisms of disease pathogenesis and development of novel treatment strategies.
Journal ArticleDOI
Clinical Characteristics and Functional Motor Outcomes of Enterovirus 71 Neurological Disease in Children.
Hooi Ling Teoh,Hooi Ling Teoh,Shekeeb S. Mohammad,Shekeeb S. Mohammad,Philip N Britton,Philip N Britton,Tejaswi Kandula,Michelle S Lorentzos,Robert Booy,Cheryl A Jones,Cheryl A Jones,William D. Rawlinson,Vidiya Ramachandran,Michael Rodriguez,P. Ian Andrews,Russell C. Dale,Russell C. Dale,Michelle A. Farrar,Michelle A. Farrar,Hugo Sampaio,Hugo Sampaio +20 more
TL;DR: Enterovirus 71 may cause serious neurological disease in young patients and the distinct clinicoradiological syndromes, predominantly within the spinal cord and brainstem, enable rapid recognition within evolving outbreaks.