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SPP1 genotype is a determinant of disease severity in Duchenne muscular dystrophy.
Pegoraro E, Hoffman EP, Piva L, Gavassini BF, Cagnin S, Ermani M, Bello L, Soraru G, Pacchioni B, Bonifati MD, Lanfranchi G, Angelini C, Kesari A, Lee I, Gordish-Dressman H, Devaney JM, McDonald CM; Cooperative International Neuromuscular Research Group. Pegoraro E, et al. Among authors: mcdonald cm. Neurology. 2011 Jan 18;76(3):219-26. doi: 10.1212/WNL.0b013e318207afeb. Epub 2010 Dec 22. Neurology. 2011. PMID: 21178099 Free PMC article.
Development of a model-based clinical trial simulation platform to optimize the design of clinical trials for Duchenne muscular dystrophy.
Lingineni K, Aggarwal V, Morales JF, Conrado DJ, Corey D, Vong C, Burton J, Larkindale J, Romero K, Schmidt S, Kim S; Cooperative International Neuromuscular Research Group investigators and Duchenne Regulatory Science Consortium members. Lingineni K, et al. CPT Pharmacometrics Syst Pharmacol. 2022 Mar;11(3):318-332. doi: 10.1002/psp4.12753. Epub 2022 Jan 3. CPT Pharmacometrics Syst Pharmacol. 2022. PMID: 34877803 Free PMC article.
Profiles of neuromuscular diseases. Spinal muscular atrophy.
Carter GT, Abresch RT, Fowler WM Jr, Johnson ER, Kilmer DD, McDonald CM. Carter GT, et al. Among authors: mcdonald cm. Am J Phys Med Rehabil. 1995 Sep-Oct;74(5 Suppl):S150-9. doi: 10.1097/00002060-199509001-00009. Am J Phys Med Rehabil. 1995. PMID: 7576422
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. Among authors: mcdonald cm. Muscle Nerve. 2014 Oct;50(4):477-87. doi: 10.1002/mus.24332. Muscle Nerve. 2014. PMID: 25042182 Free PMC article. Clinical Trial.
Peripheral neuropathies of childhood.
McDonald CM. McDonald CM. Phys Med Rehabil Clin N Am. 2001 May;12(2):473-90. Phys Med Rehabil Clin N Am. 2001. PMID: 11345019 Review.
Profiles of neuromuscular diseases. Duchenne muscular dystrophy.
McDonald CM, Abresch RT, Carter GT, Fowler WM Jr, Johnson ER, Kilmer DD, Sigford BJ. McDonald CM, et al. Am J Phys Med Rehabil. 1995 Sep-Oct;74(5 Suppl):S70-92. doi: 10.1097/00002060-199509001-00003. Am J Phys Med Rehabil. 1995. PMID: 7576424
303 results