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Academic Journal
Scala, M.; Scudieri, P.; Balagura, G.; Zara, F.; Striano, P.; Drouot, N.; Charlet-Berguerand, N.; Piton, A.; MacLennan, S.C.; Wessels, M.W.; van Slegtenhorst, M.; Saris, J.J.; Krygier, M.; Pavinato, L.; Brusco, A.; Telegrafi, A.; de Man, S.A.; Iacomino, M.; Madia, F.; Uva, P.; Giacomini, T.; Nobile, G.; Mancardi, M.M.; Galloni, G.B.; Verrotti, A.; Umair, M.; Khan, A.; Liebelt, J.; Schmidts, M.; Langer, T.; Lipska-Ziętkiewicz, B.S.
In: Human Mutation . (Human Mutation, September 2022, 43(9):1299-1313)
Onasemnogene abeparvovec in spinal muscular atrophy: an Australian experience of safety and efficacy
Academic Journal
D'Silva, A.M.; Holland, S.; Kariyawasam, D.; Sampaio, H.; Farrar, M.A.; Herbert, K.; Barclay, P.; Cairns, A.; MacLennan, S.C.; Ryan, M.M.; Woodcock, I.R.; Yiu, E.M.; Smith, N.; Alexander, I.E.
In: Annals of Clinical and Translational Neurology . (Annals of Clinical and Translational Neurology, March 2022, 9(3):339-350)
Academic Journal
Jin, S.C.; Zeng, X.; Sierant, M.C.; Zhang, J.; Sheth, A.H.; Bilguvar, K.; Lifton, R.P.; Lewis, S.A.; Bakhtiari, S.; Shetty, S.; Nordlie, S.M.; Elie, A.; Norton, B.Y.; Guida, B.S.; Magee, H.; Liu, J.; Heim, J.; Padilla-Lopez, S.; Kruer, M.C.; Corbett, M.A.; van Eyk, C.L.; Berry, J.G.; Harper, K.; Webber, D.L.; Frank, M.S.B.; Gecz, J.; MacLennan, A.H.; Haider, S.; Pastore, S.; Vincent, J.B.; Brunstrom-Hernandez, J.; Papavasileiou, A.; Fahey, M.C.; Zhou, C.; Shang, Q.; Li, B.; Xia, L.; Xu, Y.; Zhu, D.; Zhang, B.; Wang, X.; Zhu, C.; Knight, J.R.; Castaldi, C.; Tikhonova, I.R.; López-Giráldez, F.; Mane, S.M.; Keren, B.; Buratti, J.; Whalen, S.; Doummar, D.; Cho, M.; Retterer, K.; Millan, F.; Wang, Y.; Xing, Q.; Waugh, J.L.; Rodan, L.; Cohen, J.S.; Fatemi, A.; Lin, A.E.; Phillips, J.P.; Feyma, T.; MacLennan, S.C.; Vaughan, S.; Zarnescu, D.C.; Crompton, K.E.; Reid, S.M.; Reddihough, D.S.; Amor, D.J.; Gao, C.; Novak, I.; Badawi, N.; Wilson, Y.A.; McIntyre, S.J.; Lu, Q.
In: Nature Genetics . (Nature Genetics, 1 October 2020, 52(10):1046-1056)
Academic Journal
Bryen, S.J.; O'Grady, G.; Bournazos, A.M.; Cooper, S.T.; Ewans, L.J.; Pinner, J.; MacLennan, S.C.; Donkervoort, S.; Hu, Y.; Bönnemann, C.G.; Castro, D.; Töpf, A.; Straub, V.; Cummings, B.; Chao, K.R.; Weisburd, B.; Francioli, L.; MacArthur, D.G.; Faiz, F.; Davis, M.R.; Malicki, D.M.; Doyle, H.; Witting, N.; Vissing, J.; Claeys, K.G.; Majumdar, A.; Baptista, J.; Ellard, S.; Grosmann, C.; Savarese, M.; Johari, M.; Vihola, A.; Udd, B.; Beleza-Meireles, A.; Urankar, K.
In: Human Mutation . (Human Mutation, 1 February 2020, 41(2):403-411)
Academic Journal
In: JAMA Pediatrics . (JAMA Pediatrics, 1 May 2023, 177(5):455-456)
Academic Journal
Jin, S.C.; Zeng, X.; Sierant, M.C.; Zhang, J.; Sheth, A.H.; Bilguvar, K.; Lifton, R.P.; Lewis, S.A.; Bakhtiari, S.; Shetty, S.; Nordlie, S.M.; Elie, A.; Norton, B.Y.; Guida, B.S.; Magee, H.; Liu, J.; Heim, J.; Padilla-Lopez, S.; Kruer, M.C.; Corbett, M.A.; van Eyk, C.L.; Berry, J.G.; Harper, K.; Webber, D.L.; Frank, M.S.B.; Gecz, J.; MacLennan, A.H.; Haider, S.; Pastore, S.; Vincent, J.B.; Brunstrom-Hernandez, J.; Papavasileiou, A.; Fahey, M.C.; Zhou, C.; Shang, Q.; Li, B.; Zhao, H.; Xia, L.; Xu, Y.; Zhu, D.; Zhang, B.; Wang, X.; Zhu, C.; Knight, J.R.; Castaldi, C.; Tikhonova, I.R.; López-Giráldez, F.; Mane, S.M.; Keren, B.; Buratti, J.; Whalen, S.; Doummar, D.; Cho, M.; Retterer, K.; Millan, F.; Wang, Y.; Xing, Q.; Waugh, J.L.; Rodan, L.; Cohen, J.S.; Fatemi, A.; Lin, A.E.; Phillips, J.P.; Feyma, T.; MacLennan, S.C.; Vaughan, S.; Zarnescu, D.C.; Crompton, K.E.; Reid, S.M.; Reddihough, D.S.; Amor, D.J.; Gao, C.; Novak, I.; Badawi, N.; Wilson, Y.A.; McIntyre, S.J.; Lu, Q.
In: Nature Genetics . (Nature Genetics, March 2021, 53(3):412)
Academic Journal
In: Pediatric Radiology . (Pediatric Radiology, February 2008, 38(2):227-229)
Academic Journal
In: Journal of Child Neurology . (Journal of Child Neurology, 2008, 23(11):1300-1304)
Academic Journal
Pham, R.; Palmer, L.J.; Mol, B.W.; MacLennan, A.H.; Berry, J.G.; Gecz, J.; Corbett, M.A.; van Eyk, C.L.; Webber, D.L.; MacLennan, S.C.
In: Developmental Medicine and Child Neurology . (Developmental Medicine and Child Neurology, 1 September 2020, 62(9):1024-1030)
Academic Journal
In: Journal of Child Neurology . (Journal of Child Neurology, August 2004, 19(8):626-627)
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[AR] MacLennan, S.C.
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