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Effect of Different Corticosteroid Dosing Regimens on Clinical Outcomes in Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial.
JAMA. 2022 Apr 19;327(15):1456-1468. doi: 10.1001/jama.2022.4315.
JAMA. 2022.
PMID: 35381069
Free PMC article.
Clinical Trial.
Growth pattern trajectories in boys with Duchenne muscular dystrophy.
Stimpson G, Raquq S, Chesshyre M, Fewtrell M, Ridout D, Sarkozy A, Manzur A, Ayyar Gupta V, De Amicis R, Muntoni F, Baranello G; NorthStar Network.
Stimpson G, et al.
Orphanet J Rare Dis. 2022 Jan 24;17(1):20. doi: 10.1186/s13023-021-02158-9.
Orphanet J Rare Dis. 2022.
PMID: 35073949
Free PMC article.
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Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy: A 30-Month Nonrandomized Controlled Open-Label Extension Trial.
Mah JK, Clemens PR, Guglieri M, Smith EC, Finkel RS, Tulinius M, Nevo Y, Ryan MM, Webster R, Castro D, Kuntz NL, McDonald CM, Damsker JM, Schwartz BD, Mengle-Gaw LJ, Jackowski S, Stimpson G, Ridout DA, Ayyar-Gupta V, Baranello G, Manzur AY, Muntoni F, Gordish-Dressman H, Leinonen M, Ward LM, Hoffman EP, Dang UJ; NorthStar UK Network and CINRG DNHS Investigators.
Mah JK, et al.
JAMA Netw Open. 2022 Jan 4;5(1):e2144178. doi: 10.1001/jamanetworkopen.2021.44178.
JAMA Netw Open. 2022.
PMID: 35076703
Free PMC article.
Clinical Trial.
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Peak functional ability and age at loss of ambulation in Duchenne muscular dystrophy.
Zambon AA, Ayyar Gupta V, Ridout D, Manzur AY, Baranello G, Trucco F, Muntoni F; UK Northstar Clinical Network.
Zambon AA, et al.
Dev Med Child Neurol. 2022 Aug;64(8):979-988. doi: 10.1111/dmcn.15176. Epub 2022 Feb 14.
Dev Med Child Neurol. 2022.
PMID: 35385138
Free PMC article.
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A novel MYH2 mutation in family members presenting with congenital myopathy, ophthalmoplegia and facial weakness.
Willis T, Hedberg-Oldfors C, Alhaswani Z, Kulshrestha R, Sewry C, Oldfors A.
Willis T, et al. Among authors: alhaswani z.
J Neurol. 2016 Jul;263(7):1427-33. doi: 10.1007/s00415-016-8154-8. Epub 2016 May 13.
J Neurol. 2016.
PMID: 27177998
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Clinical and Genetic Characteristics in Young, Glucocorticoid-Naive Boys With Duchenne Muscular Dystrophy.
Schiava M, Amos R, VanRuiten H, McDermott MP, Martens WB, Gregory S, Mayhew A, McColl E, Tawil R, Willis T, Bushby K, Griggs RC, Guglieri M; FOR-DMD Investigators of the Muscle Study Group.
Schiava M, et al.
Neurology. 2022 Jan 24;98(4):e390-e401. doi: 10.1212/WNL.0000000000013122.
Neurology. 2022.
PMID: 34857536
Free PMC article.
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