메뉴 건너뛰기




Volumn 25, Issue 5, 2012, Pages 604-608

Pharmacological therapies for muscular dystrophies

Author keywords

muscular dystrophy; pharmacologic; therapy

Indexed keywords

AGENTS ACTING ON THE PERIPHERAL NERVOUS AND NEUROMUSCULAR SYSTEMS; ANGIOTENSIN RECEPTOR ANTAGONIST; ANTIINFLAMMATORY AGENT; DEFLAZACORT; DIPEPTIDYL CARBOXYPEPTIDASE INHIBITOR; DYSTROPHIN; MEXILETINE; NABUMETONE; PREDNISONE; SMT C1100; UNCLASSIFIED DRUG; UTROPHIN;

EID: 84866342485     PISSN: 13507540     EISSN: 14736551     Source Type: Journal    
DOI: 10.1097/WCO.0b013e328357f44c     Document Type: Review
Times cited : (21)

References (41)
  • 1
    • 16444378040 scopus 로고    scopus 로고
    • Dystrophinopathies
    • Engel AG, Franzini-Armstrong C, editors 3rd ed. New York: McGraw-Hill
    • Engel AG, Ozawa E. Dystrophinopathies. In Engel AG, Franzini-Armstrong C, editors. Myology. 3rd ed. New York: McGraw-Hill; 2004. pp. 961-1025.
    • (2004) Myology
    • Engel, A.G.1    Ozawa, E.2
  • 2
    • 0025869545 scopus 로고
    • Prednisone in Duchenne dystrophy. A randomized, controlled trial defining the time course and dose response. Clinical Investigation of Duchenne Dystrophy Group
    • Griggs RC, Moxley RT III, Mendell JR, et al. Prednisone in Duchenne dystrophy. A randomized, controlled trial defining the time course and dose response. Clinical Investigation of Duchenne Dystrophy Group. Arch Neurol 1991; 48:383-388.
    • (1991) Arch Neurol , vol.48 , pp. 383-388
    • Griggs, R.C.1    Moxley Iii., R.T.2    Mendell, J.R.3
  • 3
    • 0033812726 scopus 로고    scopus 로고
    • A multicenter, double-blind, randomized trial of deflazacort versus prednisone in Duchenne muscular dystrophy
    • Bonifati MD, Ruzza G, Bonometto P, et al. A multicenter, double-blind, randomized trial of deflazacort versus prednisone in Duchenne muscular dystrophy. Muscle Nerve 2000; 23:1344-1347.
    • (2000) Muscle Nerve , vol.23 , pp. 1344-1347
    • Bonifati, M.D.1    Ruzza, G.2    Bonometto, P.3
  • 4
    • 78650186613 scopus 로고    scopus 로고
    • Use of corticosteroids in a populationbased cohort of boys with Duchenne and Becker muscular dystrophy
    • Matthews DJ, James KA, Miller LA, et al. Use of corticosteroids in a populationbased cohort of boys with Duchenne and Becker muscular dystrophy. J Child Neurol 2010; 25:1319-1324.
    • (2010) J Child Neurol , vol.25 , pp. 1319-1324
    • Matthews, D.J.1    James, K.A.2    Miller, L.A.3
  • 5
    • 80051500586 scopus 로고    scopus 로고
    • Randomized, blinded trial of weekend vs. daily prednisone in Duchenne muscular dystrophy
    • Escolar DM, Hache LP, Clemens PR, et al. Randomized, blinded trial of weekend vs. daily prednisone in Duchenne muscular dystrophy. Neurology 2011; 77:444-452.
    • (2011) Neurology , vol.77 , pp. 444-452
    • Escolar, D.M.1    Hache, L.P.2    Clemens, P.R.3
  • 6
    • 79959297748 scopus 로고    scopus 로고
    • Cardiomyopathy of Duchenne muscular dystrophy: Current understanding and future directions
    • Spurney CF. Cardiomyopathy of Duchenne muscular dystrophy: current understanding and future directions. Muscle Nerve 2011; 44:8-19.
    • (2011) Muscle Nerve , vol.44 , pp. 8-19
    • Spurney, C.F.1
  • 7
    • 79951479823 scopus 로고    scopus 로고
    • Losartan decreases cardiac muscle fibrosis and improves cardiac function in dystrophin-deficient mdx mice
    • Spurney CF, Sali A, Guerron AD, et al. Losartan decreases cardiac muscle fibrosis and improves cardiac function in dystrophin-deficient mdx mice. J Cardiovasc Pharmacol Ther 2011; 16:87-95.
    • (2011) J Cardiovasc Pharmacol Ther , vol.16 , pp. 87-95
    • Spurney, C.F.1    Sali, A.2    Guerron, A.D.3
  • 8
    • 84857668964 scopus 로고    scopus 로고
    • Effects of steroids and angiotensin converting enzyme inhibition on circumferential strain in boys with Duchenne muscular dystrophy: A cross-sectional and longitudinal study utilizing cardiovascular magnetic resonance
    • Hor KN, Mazur W, Taylor MD, et al. Effects of steroids and angiotensin converting enzyme inhibition on circumferential strain in boys with Duchenne muscular dystrophy: a cross-sectional and longitudinal study utilizing cardiovascular magnetic resonance. J Cardiovasc Magn Reson 2011; 13:60.
    • (2011) J Cardiovasc Magn Reson , vol.13 , pp. 60
    • Hor, K.N.1    Mazur, W.2    Taylor, M.D.3
  • 9
    • 84858338127 scopus 로고    scopus 로고
    • Nitric oxide donor and nonsteroidal anti-inflammatory drugs as a therapy for muscular dystrophies: Evidence from a safety study with pilot efficacy measures in adult dystrophic patients
    • D'Angelo MG, Gandossini S, Martinelli BF, et al. Nitric oxide donor and nonsteroidal anti-inflammatory drugs as a therapy for muscular dystrophies: evidence from a safety study with pilot efficacy measures in adult dystrophic patients. Pharmacol Res 2012; 65:472-479.
    • (2012) Pharmacol Res , vol.65 , pp. 472-479
    • D'Angelo, M.G.1    Gandossini, S.2    Martinelli, B.F.3
  • 10
    • 0025932274 scopus 로고
    • Immunolocalization and developmental expression of dystrophin related protein in skeletal muscle
    • Khurana TS, Watkins SC, Chafey P, et al. Immunolocalization and developmental expression of dystrophin related protein in skeletal muscle. Neuromuscul Disord 1991; 1:185-194.
    • (1991) Neuromuscul Disord , vol.1 , pp. 185-194
    • Khurana, T.S.1    Watkins, S.C.2    Chafey, P.3
  • 13
    • 79551667507 scopus 로고    scopus 로고
    • Biglycan recruits utrophin to the sarcolemma and counters dystrophic pathology in mdx mice
    • Amenta AR, Yilmaz A, Bogdanovich S, et al. Biglycan recruits utrophin to the sarcolemma and counters dystrophic pathology in mdx mice. Proc Natl Acad Sci USA 2011; 108:762-767.
    • (2011) Proc Natl Acad Sci USA , vol.108 , pp. 762-767
    • Amenta, A.R.1    Yilmaz, A.2    Bogdanovich, S.3
  • 14
    • 79955856839 scopus 로고    scopus 로고
    • Discovery of 2-arylbenzoxazoles as upregulators of utrophin production for the treatment of Duchenne muscular dystrophy
    • Chancellor DR, Davies KE, De MO, et al. Discovery of 2-arylbenzoxazoles as upregulators of utrophin production for the treatment of Duchenne muscular dystrophy. J Med Chem 2011; 54:3241-3250.
    • (2011) J Med Chem , vol.54 , pp. 3241-3250
    • Chancellor, D.R.1    Davies, K.E.2    De, M.O.3
  • 15
    • 79960918733 scopus 로고    scopus 로고
    • Discovery and SAR of 2-arylbenzotriazoles and 2-arylindazoles as potential treatments for Duchenne muscular dystrophy
    • De MO, Dorgan CR, Johnson PD, et al. Discovery and SAR of 2-arylbenzotriazoles and 2-arylindazoles as potential treatments for Duchenne muscular dystrophy. Bioorg Med Chem Lett 2011; 21:4828-4831.
    • (2011) Bioorg Med Chem Lett , vol.21 , pp. 4828-4831
    • De Mo Dorgan, C.R.1    Johnson, P.D.2
  • 16
    • 79955867741 scopus 로고    scopus 로고
    • Daily treatment with SMTC1100, a novel small molecule utrophin upregulator, dramatically reduces the dystrophic symptoms in the mdx mouse
    • Tinsley JM, Fairclough RJ, Storer R, et al. Daily treatment with SMTC1100, a novel small molecule utrophin upregulator, dramatically reduces the dystrophic symptoms in the mdx mouse. PLoS One 2011; 6:e19189.
    • (2011) PLoS One , vol.6
    • Tinsley, J.M.1    Fairclough, R.J.2    Storer, R.3
  • 17
    • 80054779433 scopus 로고    scopus 로고
    • Drug discovery for Duchenne muscular dystrophy via utrophin promoter activation screening
    • Moorwood C, Lozynska O, Suri N, et al. Drug discovery for Duchenne muscular dystrophy via utrophin promoter activation screening. PLoS One 2011; 6:e26169.
    • (2011) PLoS One , vol.6
    • Moorwood, C.1    Lozynska, O.2    Suri, N.3
  • 18
    • 84455172952 scopus 로고    scopus 로고
    • Translational regulation of utrophin by miRNAs
    • Basu U, Lozynska O, Moorwood C, et al. Translational regulation of utrophin by miRNAs. PLoS One 2011; 6:e29376.
    • (2011) PLoS One , vol.6
    • Basu, U.1    Lozynska, O.2    Moorwood, C.3
  • 19
    • 33746766278 scopus 로고    scopus 로고
    • Entries in the Leiden Duchenne muscular dystrophy mutation database: An overview of mutation types and paradoxical cases that confirm the reading-frame rule
    • DOI 10.1002/mus.20586
    • artsma-Rus A, van Deutekom JC, Fokkema IF, et al. Entries in the Leiden Duchenne muscular dystrophy mutation database: an overview of mutation types and paradoxical cases that confirm the reading-frame rule. Muscle Nerve 2006; 34:135-144. (Pubitemid 44167796)
    • (2006) Muscle and Nerve , vol.34 , Issue.2 , pp. 135-144
    • Aartsma-Rus, A.1    Van Deutekom, J.C.T.2    Fokkema, I.F.3    Van Ommen, G.-J.B.4    Den Dunnen, J.T.5
  • 20
    • 77952938084 scopus 로고    scopus 로고
    • Gentamicin-induced readthrough of stop codons inDuchennemuscular dystrophy
    • Malik V, Rodino-Klapac LR, Viollet L, et al. Gentamicin-induced readthrough of stop codons inDuchennemuscular dystrophy.AnnNeurol2010; 67:771-780.
    • (2010) AnnNeurol , vol.67 , pp. 771-780
    • Malik, V.1    Rodino-Klapac, L.R.2    Viollet, L.3
  • 23
    • 77956311645 scopus 로고    scopus 로고
    • Read-through strategies for suppression of nonsense mutations in Duchenne/Becker muscular dystrophy: Aminoglycosides and ataluren (PTC124)
    • Finkel RS. Read-through strategies for suppression of nonsense mutations in Duchenne/Becker muscular dystrophy: aminoglycosides and ataluren (PTC124). J Child Neurol 2010; 25:1158-1164.
    • (2010) J Child Neurol , vol.25 , pp. 1158-1164
    • Finkel, R.S.1
  • 26
    • 0017646399 scopus 로고
    • Fractures of long bones in duchenne muscular dystrophy
    • Siegel IM. Fractures of long bones in Duchenne muscular dystrophy. J Trauma 1977; 17:219-222. (Pubitemid 8096954)
    • (1977) Journal of Trauma , vol.17 , Issue.3 , pp. 219-222
    • Siegel, I.M.1
  • 28
    • 72149108443 scopus 로고    scopus 로고
    • Diagnosis and management of Duchenne muscular dystrophy, part 1: Diagnosis, and pharmacological and psychosocial management
    • Bushby K, Finkel R, Birnkrant DJ, et al. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management. Lancet Neurol 2010; 9:77-93.
    • (2010) Lancet Neurol , vol.9 , pp. 77-93
    • Bushby, K.1    Finkel, R.2    Birnkrant, D.J.3
  • 29
    • 76549130473 scopus 로고    scopus 로고
    • Diagnosis and management of Duchenne muscular dystrophy, part 2: Implementation of multidisciplinary care
    • Bushby K, Finkel R, Birnkrant DJ, et al. Diagnosis and management of Duchenne muscular dystrophy, part 2: implementation of multidisciplinary care. Lancet Neurol 2010; 9:177-189.
    • (2010) Lancet Neurol , vol.9 , pp. 177-189
    • Bushby, K.1    Finkel, R.2    Birnkrant, D.J.3
  • 31
    • 0036166717 scopus 로고    scopus 로고
    • Vertebral compression in Duchenne muscular dystrophy following deflazacort
    • DOI 10.1016/S0960-8966(01)00307-8, PII S0960896601003078
    • Talim B, Malaguti C, Gnudi S, et al. Vertebral compression in Duchenne muscular dystrophy following deflazacort. Neuromuscul Disord 2002; 12:294-295. (Pubitemid 34136914)
    • (2002) Neuromuscular Disorders , vol.12 , Issue.3 , pp. 294-295
    • Talim, B.1    Malaguti, C.2    Gnudi, S.3    Politano, L.4    Merlini, L.5
  • 32
    • 84861705331 scopus 로고    scopus 로고
    • Bisphosphonates for osteoporosis: Where do we go from here?
    • Whitaker M, Guo J, Kehoe T, Benson G. Bisphosphonates for osteoporosis: where do we go from here? N Engl J Med 2012; 366:2048-2051.
    • (2012) N Engl J Med , vol.366 , pp. 2048-2051
    • Whitaker, M.1    Guo, J.2    Kehoe, T.3    Benson, G.4
  • 33
    • 84868208578 scopus 로고    scopus 로고
    • The use of intravenous bisphosphonate therapy to treat vertebral fractures due to osteoporosis among boys with Duchenne muscular dystrophy
    • [Epub ahead of print]
    • Sbrocchi AM, Rauch F, Jacob P, et al. The use of intravenous bisphosphonate therapy to treat vertebral fractures due to osteoporosis among boys with Duchenne muscular dystrophy. Osteoporos Int 2012. [Epub ahead of print]
    • (2012) Osteoporos Int
    • Sbrocchi, A.M.1    Rauch, F.2    Jacob, P.3
  • 34
    • 79551545969 scopus 로고    scopus 로고
    • Impact of bisphosphonates on survival for patients with Duchenne muscular dystrophy
    • Gordon KE, Dooley JM, Sheppard KM, et al. Impact of bisphosphonates on survival for patients with Duchenne muscular dystrophy. Pediatrics 2011; 127:e353-e358.
    • (2011) Pediatrics , vol.127
    • Gordon, K.E.1    Dooley, J.M.2    Sheppard, K.M.3
  • 36
    • 33748854083 scopus 로고    scopus 로고
    • Myotonic dystrophy
    • Engel AG, Franzini-Armstrong C, editors 3rd ed. New York: McGraw-Hill
    • Harper PS, Monckton DG. Myotonic dystrophy. In Engel AG, Franzini-Armstrong C, editors. Myology. 3rd ed. New York: McGraw-Hill; 2004. pp. 1039-1076.
    • (2004) Myology , pp. 1039-1076
    • Harper, P.S.1    Monckton, D.G.2
  • 37
    • 84859521683 scopus 로고    scopus 로고
    • Myotonic dystrophy type 1 or Steinert's disease
    • Romeo V. Myotonic dystrophy type 1 or Steinert's disease. Adv Exp Med Biol 2012; 724:239-257.
    • (2012) Adv Exp Med Biol , vol.724 , pp. 239-257
    • Romeo, V.1
  • 38
    • 77951848201 scopus 로고    scopus 로고
    • Mexiletine is an effective antimyotonia treatment in myotonic dystrophy type 1
    • Logigian EL, Martens WB, Moxley RT, et al. Mexiletine is an effective antimyotonia treatment in myotonic dystrophy type 1. Neurology 2010; 74:1441-1448.
    • (2010) Neurology , vol.74 , pp. 1441-1448
    • Logigian, E.L.1    Martens, W.B.2    Moxley, R.T.3
  • 39
    • 33644510962 scopus 로고    scopus 로고
    • Facioscapulohumeral muscular dystrophy and scapuloperoneal disorders
    • Engel AG, Franzini-Armstrong C, editors 3rd ed. New York: McGraw-Hill
    • Flanigan KM. Facioscapulohumeral muscular dystrophy and scapuloperoneal disorders. In Engel AG, Franzini-Armstrong C, editors. Myology. 3rd ed. New York: McGraw-Hill; 2004. pp. 1123-1133.
    • (2004) Myology , pp. 1123-1133
    • Flanigan, K.M.1
  • 40
    • 79957512849 scopus 로고    scopus 로고
    • The muscular dystrophies: Distinct pathogenic mechanisms invite novel therapeutic approaches
    • Sahenk Z, Mendell Jr. The muscular dystrophies: distinct pathogenic mechanisms invite novel therapeutic approaches. Curr Rheumatol Rep 2011; 13:199-207.
    • (2011) Curr Rheumatol Rep , vol.13 , pp. 199-207
    • Sahenk, Z.1    Mendell, J.R.2
  • 41
    • 84856255633 scopus 로고    scopus 로고
    • Changes in pain-related beliefs, coping, and catastrophizing predict changes in pain intensity, pain interference, and psychological functioning in individuals with myotonic muscular dystrophy and facioscapulohumeral dystrophy
    • Nieto R, Raichle KA, Jensen MP, Miro J. Changes in pain-related beliefs, coping, and catastrophizing predict changes in pain intensity, pain interference, and psychological functioning in individuals with myotonic muscular dystrophy and facioscapulohumeral dystrophy. Clin J Pain 2012; 28:47-54.
    • (2012) Clin J Pain , vol.28 , pp. 47-54
    • Nieto, R.1    Raichle, K.A.2    Jensen, M.P.3    Miro, J.4


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.