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Patient-reported impact of symptoms in adrenoleukodystrophy (PRISM-ALD).
Varma A, Weinstein J, Seabury J, Rosero S, Dilek N, Heatwole J, Engebrecht C, Khosa S, Chung K, Paker A, Woo A, Brooks G, Beals C, Gandhi R, Heatwole C. Varma A, et al. Among authors: heatwole c. Orphanet J Rare Dis. 2024 Mar 19;19(1):127. doi: 10.1186/s13023-024-03129-6. Orphanet J Rare Dis. 2024. PMID: 38504253 Free PMC article.
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. Muscle Nerve. 2014 Oct;50(4):477-87. doi: 10.1002/mus.24332. Muscle Nerve. 2014. PMID: 25042182 Free PMC article. Clinical Trial.
Brief assessment of cognitive function in myotonic dystrophy: Multicenter longitudinal study using computer-assisted evaluation.
Deutsch GK, Hagerman KA, Sampson J, Dent G, Dekdebrun J, Parker DM, Thornton CA, Heatwole CR, Subramony SH, Mankodi AK, Ashizawa T, Statland JM, Arnold WD, Moxley RT 3rd, Day JW; Myotonic Dystrophy Clinical Research Network. Deutsch GK, et al. Muscle Nerve. 2022 May;65(5):560-567. doi: 10.1002/mus.27520. Epub 2022 Feb 28. Muscle Nerve. 2022. PMID: 35179228 Free PMC article.
Open-label trial of recombinant human insulin-like growth factor 1/recombinant human insulin-like growth factor binding protein 3 in myotonic dystrophy type 1.
Heatwole CR, Eichinger KJ, Friedman DI, Hilbert JE, Jackson CE, Logigian EL, Martens WB, McDermott MP, Pandya SK, Quinn C, Smirnow AM, Thornton CA, Moxley RT 3rd. Heatwole CR, et al. Arch Neurol. 2011 Jan;68(1):37-44. doi: 10.1001/archneurol.2010.227. Epub 2010 Sep 13. Arch Neurol. 2011. PMID: 20837825 Free PMC article. Clinical Trial.
The nondystrophic myotonias.
Heatwole CR, Moxley RT 3rd. Heatwole CR, et al. Neurotherapeutics. 2007 Apr;4(2):238-51. doi: 10.1016/j.nurt.2007.01.012. Neurotherapeutics. 2007. PMID: 17395134 Free article. Review.
94 results