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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.
A multi-center screening trial of rasagiline in patients with amyotrophic lateral sclerosis: Possible mitochondrial biomarker target engagement.
Macchi Z, Wang Y, Moore D, Katz J, Saperstein D, Walk D, Simpson E, Genge A, Bertorini T, Fernandes JA, Swenson A, Elman L, Dimachkie M, Herbelin L, Miller J, Lu J, Wilkins H, Swerdlow RH, Statland J, Barohn R; Western ALS (WALS) Rasagiline Study Group. Macchi Z, et al. Amyotroph Lateral Scler Frontotemporal Degener. 2015;16(5-6):345-52. doi: 10.3109/21678421.2015.1026826. Epub 2015 Apr 2. Amyotroph Lateral Scler Frontotemporal Degener. 2015. PMID: 25832828 Free PMC article.