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Page 1
Psychometric properties of the PEDI-CAT for children and youth with spinal muscular atrophy.
J Pediatr Rehabil Med. 2021;14(3):451-461. doi: 10.3233/PRM-190664.
J Pediatr Rehabil Med. 2021.
PMID: 34275913
Ataluren treatment of patients with nonsense mutation dystrophinopathy.
Bushby K, Finkel R, Wong B, Barohn R, Campbell C, Comi GP, Connolly AM, Day JW, Flanigan KM, Goemans N, Jones KJ, Mercuri E, Quinlivan R, Renfroe JB, Russman B, Ryan MM, Tulinius M, Voit T, Moore SA, Lee Sweeney H, Abresch RT, Coleman KL, Eagle M, Florence J, Gappmaier E, Glanzman AM, Henricson E, Barth J, Elfring GL, Reha A, Spiegel RJ, O'donnell MW, Peltz SW, Mcdonald CM; PTC124-GD-007-DMD STUDY GROUP.
Bushby K, et al.
Muscle Nerve. 2014 Oct;50(4):477-87. doi: 10.1002/mus.24332.
Muscle Nerve. 2014.
PMID: 25042182
Free PMC article.
Clinical Trial.
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Spinal muscular atrophy functional composite score: A functional measure in spinal muscular atrophy.
Montes J, Glanzman AM, Mazzone ES, Martens WB, Dunaway S, Pasternak A, Riley SO, Quigley J, Pandya S, De Vivo DC, Kaufmann P, Chiriboga CA, Finkel RS, Tennekoon GI, Darras BT, Pane M, Mercuri E, Mcdermott MP; Pediatric Neuromuscular Clinical Research Network, Muscle Study Group, SMA Europe.
Montes J, et al. Among authors: riley so.
Muscle Nerve. 2015 Dec;52(6):942-7. doi: 10.1002/mus.24670. Epub 2015 Oct 10.
Muscle Nerve. 2015.
PMID: 25846132
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Motor and cognitive assessment of infants and young boys with Duchenne Muscular Dystrophy: results from the Muscular Dystrophy Association DMD Clinical Research Network.
Connolly AM, Florence JM, Cradock MM, Malkus EC, Schierbecker JR, Siener CA, Wulf CO, Anand P, Golumbek PT, Zaidman CM, Philip Miller J, Lowes LP, Alfano LN, Viollet-Callendret L, Flanigan KM, Mendell JR, McDonald CM, Goude E, Johnson L, Nicorici A, Karachunski PI, Day JW, Dalton JC, Farber JM, Buser KK, Darras BT, Kang PB, Riley SO, Shriber E, Parad R, Bushby K, Eagle M; MDA DMD Clinical Research Network.
Connolly AM, et al. Among authors: riley so.
Neuromuscul Disord. 2013 Jul;23(7):529-39. doi: 10.1016/j.nmd.2013.04.005. Epub 2013 May 28.
Neuromuscul Disord. 2013.
PMID: 23726376
Free PMC article.
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One year outcome of boys with Duchenne muscular dystrophy using the Bayley-III scales of infant and toddler development.
Connolly AM, Florence JM, Cradock MM, Eagle M, Flanigan KM, McDonald CM, Karachunski PI, Darras BT, Bushby K, Malkus EC, Golumbek PT, Zaidman CM, Miller JP, Mendell JR; MDA DMD Clinical Research Network.
Connolly AM, et al.
Pediatr Neurol. 2014 Jun;50(6):557-63. doi: 10.1016/j.pediatrneurol.2014.02.006. Epub 2014 Feb 15.
Pediatr Neurol. 2014.
PMID: 24842254
Free PMC article.
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