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Genetic modifiers of ambulation in the Cooperative International Neuromuscular Research Group Duchenne Natural History Study.
Bello L, Kesari A, Gordish-Dressman H, Cnaan A, Morgenroth LP, Punetha J, Duong T, Henricson EK, Pegoraro E, McDonald CM, Hoffman EP; Cooperative International Neuromuscular Research Group Investigators. Bello L, et al. Ann Neurol. 2015 Apr;77(4):684-96. doi: 10.1002/ana.24370. Epub 2015 Mar 13. Ann Neurol. 2015. PMID: 25641372 Free PMC article.
Developing a Natural History Model for Duchenne Muscular Dystrophy.
Broomfield J, Hill M, Chandler F, Crowther MJ, Godfrey J, Guglieri M, Hastie J, Larkindale J, Mumby-Croft J, Reuben E, Woodcock F, Abrams KR; Project HERCULES, the Cooperative International Neuromuscular Research Group investigators, Duchenne Regulatory Science Consortium members. Broomfield J, et al. Pharmacoecon Open. 2024 Jan;8(1):79-89. doi: 10.1007/s41669-023-00450-x. Epub 2023 Nov 29. Pharmacoecon Open. 2024. PMID: 38019449 Free PMC article.
CINRG pilot trial of coenzyme Q10 in steroid-treated Duchenne muscular dystrophy.
Spurney CF, Rocha CT, Henricson E, Florence J, Mayhew J, Gorni K, Pasquali L, Pestronk A, Martin GR, Hu F, Nie L, Connolly AM, Escolar DM; Cooperative International Neuromuscular Research Group Investigators. Spurney CF, et al. Muscle Nerve. 2011 Aug;44(2):174-8. doi: 10.1002/mus.22047. Epub 2011 Jun 22. Muscle Nerve. 2011. PMID: 21698649 Free PMC article. Clinical Trial.
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.
Modeling the multi-state natural history of rare diseases with heterogeneous individual patient data: A simulation study.
Broomfield J, Abrams KR, Freeman S, Latimer N, Rutherford MJ, Crowther MJ; Project HERCULES, the Cooperative International Neuromuscular Research Group investigators and Duchenne Regulatory Science Consortium members. Broomfield J, et al. Stat Med. 2024 Jan 15;43(1):184-200. doi: 10.1002/sim.9949. Epub 2023 Nov 6. Stat Med. 2024. PMID: 37932874