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A randomized, double-blind trial of lisinopril and losartan for the treatment of cardiomyopathy in duchenne muscular dystrophy.
Allen HD, Flanigan KM, Thrush PT, Dvorchik I, Yin H, Canter C, Connolly AM, Parrish M, McDonald CM, Braunlin E, Colan SD, Day J, Darras B, Mendell JR. Allen HD, et al. Among authors: darras b. PLoS Curr. 2013 Dec 12;5:ecurrents.md.2cc69a1dae4be7dfe2bcb420024ea865. doi: 10.1371/currents.md.2cc69a1dae4be7dfe2bcb420024ea865. PLoS Curr. 2013. PMID: 24459612 Free PMC article.
A slowly progressive form of limb-girdle muscular dystrophy type 2C associated with founder mutation in the SGCG gene in Puerto Rican Hispanics.
Al-Zaidy SA, Malik V, Kneile K, Rosales XQ, Gomez AM, Lewis S, Hashimoto S, Gastier-Foster J, Kang P, Darras B, Kunkel L, Carlo J, Sahenk Z, Moore SA, Pyatt R, Mendell JR. Al-Zaidy SA, et al. Among authors: darras b. Mol Genet Genomic Med. 2015 Mar;3(2):92-8. doi: 10.1002/mgg3.125. Epub 2015 Jan 8. Mol Genet Genomic Med. 2015. PMID: 25802879 Free PMC article.
Towards regulatory endorsement of drug development tools to promote the application of model-informed drug development in Duchenne muscular dystrophy.
Conrado DJ, Larkindale J, Berg A, Hill M, Burton J, Abrams KR, Abresch RT, Bronson A, Chapman D, Crowther M, Duong T, Gordish-Dressman H, Harnisch L, Henricson E, Kim S, McDonald CM, Schmidt S, Vong C, Wang X, Wong BL, Yong F, Romero K; Duchenne Muscular Dystrophy Regulatory Science Consortium (D-RSC). Conrado DJ, et al. J Pharmacokinet Pharmacodyn. 2019 Oct;46(5):441-455. doi: 10.1007/s10928-019-09642-7. Epub 2019 May 24. J Pharmacokinet Pharmacodyn. 2019. PMID: 31127458
SMA-MAP: a plasma protein panel for spinal muscular atrophy.
Kobayashi DT, Shi J, Stephen L, Ballard KL, Dewey R, Mapes J, Chung B, McCarthy K, Swoboda KJ, Crawford TO, Li R, Plasterer T, Joyce C; Biomarkers for Spinal Muscular Atrophy Study Group; Chung WK, Kaufmann P, Darras BT, Finkel RS, Sproule DM, Martens WB, McDermott MP, De Vivo DC; Pediatric Neuromuscular Clinical Research Network; Walker MG, Chen KS. Kobayashi DT, et al. Among authors: darras bt. PLoS One. 2013;8(4):e60113. doi: 10.1371/journal.pone.0060113. Epub 2013 Apr 2. PLoS One. 2013. PMID: 23565191 Free PMC article.
Revised upper limb module for spinal muscular atrophy: 12 month changes.
Pera MC, Coratti G, Mazzone ES, Montes J, Scoto M, De Sanctis R, Main M, Mayhew A, Muni Lofra R, Dunaway Young S, Glanzman AM, Duong T, Pasternak A, Ramsey D, Darras B, Day JW, Finkel RS, De Vivo DC, Sormani MP, Bovis F, Straub V, Muntoni F, Pane M, Mercuri E; iSMAC Consortium Group. Pera MC, et al. Among authors: darras b. Muscle Nerve. 2019 Apr;59(4):426-430. doi: 10.1002/mus.26419. Epub 2019 Feb 7. Muscle Nerve. 2019. PMID: 30677148
Development of an academic disease registry for spinal muscular atrophy.
Mercuri E, Finkel R, Scoto M, Hall S, Eaton S, Rashid A, Balashkina J, Coratti G, Pera MC, Samsuddin S, Civitello M, Muntoni F; iSMAC Group. Mercuri E, et al. Neuromuscul Disord. 2019 Oct;29(10):794-799. doi: 10.1016/j.nmd.2019.08.014. Epub 2019 Aug 29. Neuromuscul Disord. 2019. PMID: 31558335
2-Year Change in Revised Hammersmith Scale Scores in a Large Cohort of Untreated Paediatric Type 2 and 3 SMA Participants.
Stimpson G, Ramsey D, Wolfe A, Mayhew A, Scoto M, Baranello G, Muni Lofra R, Main M, Milev E, Coratti G, Pane M, Sansone V, D'Amico A, Bertini E, Messina S, Bruno C, Albamonte E, Mazzone ES, Montes J, Glanzman AM, Zolkipli-Cunningham Z, Pasternak A, Duong T, Dunaway Young S, Civitello M, Marini-Bettolo C, Day JW, Darras BT, De Vivo DC, Finkel RS, Mercuri E, Muntoni F; International SMA Consortium (iSMAc). Stimpson G, et al. J Clin Med. 2023 Feb 28;12(5):1920. doi: 10.3390/jcm12051920. J Clin Med. 2023. PMID: 36902710 Free PMC article.
305 results