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Titolo:
Novel approaches to treat muscular dystrophies
Autore:
Walter, MC; Lochmuller, H;
Indirizzi:
Univ Munich, Friedrich Baur Inst, Dept Neurol, D-80336 Munich, Germany Univ Munich Munich Germany D-80336 Dept Neurol, D-80336 Munich, Germany Univ Munich, Geozentrum Munchen, D-80336 Munich, Germany Univ Munich Munich Germany D-80336 trum Munchen, D-80336 Munich, Germany
Titolo Testata:
EXPERT OPINION ON INVESTIGATIONAL DRUGS
fascicolo: 4, volume: 10, anno: 2001,
pagine: 695 - 707
SICI:
1354-3784(200104)10:4<695:NATTMD>2.0.ZU;2-Z
Fonte:
ISI
Lingua:
ENG
Soggetto:
GROWTH-FACTOR-I; ORAL CREATINE SUPPLEMENTATION; MDX MICE; MYOTONIC-DYSTROPHY; SKELETAL-MUSCLE; GENE-THERAPY; MYOBLAST TRANSFER; CONTROLLED TRIAL; DOUBLE-BLIND; CARDIAC INVOLVEMENT;
Keywords:
beta-2 adrenergic agonist; aminoglycoside antibiotic; cell therapy; dietary supplement; gene therapy; growth factor; muscular dystrophy; pharmacological therapy; physiotherapy; rehabilitation; steroid; supportive care;
Tipo documento:
Review
Natura:
Periodico
Settore Disciplinare:
Life Sciences
Citazioni:
137
Recensione:
Indirizzi per estratti:
Indirizzo: Walter, MC Univ Munich, Friedrich Baur Inst, Dept Neurol, Ziemssenstr 1A, D-80336 Munich, Germany Univ Munich Ziemssenstr 1A Munich Germany D-80336 ich, Germany
Citazione:
M.C. Walter e H. Lochmuller, "Novel approaches to treat muscular dystrophies", EXPERT OP I, 10(4), 2001, pp. 695-707

Abstract

Muscular dystrophies (MD) are a clinically and generically heterogeneous group of skeletal muscle-wasting diseases. Mutations in the dystrophin gene result in dystrophin deficiency, which constitutes the pathogenic basis of Duchenne and Becker MD (DMD and BMD). Several MD are caused by mutations inother recently identified genes coding for proteins linked to the sarcolemma, the nuclear envelope or the contractile apparatus. In addition, severalMD have been mapped to different chromosomal loci and for most of them, the identification of the molecular defect is underway. The immediate result is an ongoing reclassification of the MD into disorders defined not by clinical characteristics but specific genetic mutations. At present, therapy ofMn is based on symptomatic treatment and supportive care. Convincing evidence for clinical efficacy is only available for corticosteroids that also suffer from frequent and severe side effects. Up to now, curative therapy isnot available, although promising new molecular therapies are under investigation in animal models of MD. Current treatment strategies are discussed and a perspective for effective molecular therapy is given.

ASDD Area Sistemi Dipartimentali e Documentali, Università di Bologna, Catalogo delle riviste ed altri periodici
Documento generato il 05/04/20 alle ore 20:24:14