143:(DMD). DMD is usually discovered in early childhood and is most often seen occurring in males. There are several associated symptoms that can be observed in such patients including but not limited to a delay in walking and sitting, difficulty in breathing and heart failure. These symptoms are found as a result of the inability to synthesize dystrophin and associate protein complexes that leave muscles weak and unable to repair any damaged sustained. These perpetually weak muscles prohibit normal physical activity.
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152:
so to facilitate normal physical activity and is recommended to begin as early as possible after diagnosis. Contracture intervention is recommended for patients in the middle ambulatory stage. However, surgical approach to DMD is declining as less invasive treatment becomes available. While treatment for DMD has been observed to improve muscle function and quality of life, a cure to the debilitating disease remains to be found.
151:
There has been extensive research to discover treatment for DMD. The most common drug treated against DMD is known to be
Deflazacort yielding the greatest benefits with the most acceptable side effects. Physical therapy consists of varying exercises that aim to increase muscle strength and durability
700:
Mendell, Jerry R.; Sahenk, Zarife; Lehman, Kelly; Nease, Carrie; Lowes, Linda P.; Miller, Natalie F.; Iammarino, Megan A.; Alfano, Lindsay N.; Nicholl, Amanda; Al-Zaidy, Samiah; Lewis, Sarah; Church, Kathleen; Shell, Richard; Cripe, Linda H.; Potter, Rachael A.; Griffin, Danielle A.; Pozsgai, Eric;
139:
Muscular dystrophy, the result of mutations in the genes that encode for dystrophin and the associated proteins that binds to it can arise in various forms. The most common form is known as
703:"Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled Trial"
795:
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687:"Dec 16,2021 | Chugai In-Licenses Gene Therapy Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy | News"
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Welling PA (2008-01-01). "Scaffolding
Proteins in Transport Regulation". In Alpern RJ, Herbert SC (eds.).
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Passamano L, Taglia A, Palladino A, Viggiano E, D'Ambrosio P, Scutifero M, et al. (October 2012).
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812:
347:
Binder DK (2017-01-01). "Water
Homeostasis Dysfunction in Epilepsy". In Badaut J, Plesnila N (eds.).
65:
25:
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596:"The burden, epidemiology, costs and treatment for Duchenne muscular dystrophy: an evidence review"
76:
374:
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496:"Improvement of survival in Duchenne Muscular Dystrophy: retrospective analysis of 835 patients"
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Kirschner J, LochmĂĽller H (2011-01-01). "Sarcoglycanopathies". In Griggs RC, Amato AA (eds.).
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Ryder S, Leadley RM, Armstrong N, Westwood M, de Kock S, Butt T, et al. (April 2017).
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are associated with disorders of the dystrophin-associated protein complex.
117:
37:
738:
672:
631:
580:
537:"The Dystrophin Complex: Structure, Function, and Implications for Therapy"
521:
477:
425:
333:
310:. Handbook of Clinical Neurology. Vol. 101. Elsevier. pp. 41–46.
213:
1390:
1361:
1321:
1126:
760:"Delandistrogene moxeparvovec - Roche/Sarepta Therapeutics - AdisInsight"
701:
Dugar, Ashish; Hogan, Mark; Rodino-Klapac, Louise R. (1 September 2020).
552:
91:
653:. The Muscular Dystrophies: Molecular Basis and Therapeutic Strategies.
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Biochimica et
Biophysica Acta (BBA) - Molecular Basis of Disease
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100:
may be connected to the dystrophin-associated protein complex.
108:
The dystrophin-associated protein complex is important for
36:. It is one of the two protein complexes that make up the
255:
CHAPTER 12 - Scaffolding
Proteins in Transport Regulation
647:"Current treatment of adult Duchenne muscular dystrophy"
188:"The development of the myotendinous junction. A review"
71:
The dystrophin-associated protein complex also contains
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1190:
1152:
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881:
872:
858:
847:
836:
811:
444:"The muscular dystrophies: from genes to therapies"
56:The dystrophin-associated protein complex includes
442:Lovering RM, Porter NC, Bloch RJ (December 2005).
186:Charvet B, Ruggiero F, Le Guellec D (April 2012).
116:. It is one of two protein complexes found in the
645:Wagner KR, Lechtzin N, Judge DP (February 2007).
351:. San Diego: Academic Press. pp. 315–335.
257:. San Diego: Academic Press. pp. 325–341.
1470:
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8:
402:"The dystrophin-associated protein complex"
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878:
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22:dystrophin-associated glycoprotein complex
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232:at the U.S. National Library of Medicine
203:
400:Ehmsen J, Poon E, Davies K (July 2002).
178:
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372:
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278:
192:Muscles, Ligaments and Tendons Journal
489:
487:
437:
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230:Dystrophin-Associated+Protein+Complex
18:dystrophin-associated protein complex
7:
1431:
1429:
1182:Connective tissue in skeletal muscle
248:
246:
244:
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225:
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60:. Dystrophin binds to actin of the
1449:. You can help Knowledge (XXG) by
357:10.1016/b978-0-12-803196-4.00017-5
316:10.1016/b978-0-08-045031-5.00003-7
90:, dystrophin may be replaced with
79:, which is fixed to dystrophin in
14:
600:Orphanet Journal of Rare Diseases
1433:
535:Gao QQ, McNally EM (July 2015).
263:10.1016/b978-012088488-9.50015-2
1213:Excitation–contraction coupling
75:. This includes a four subunit
46:integrin-vinculin-talin complex
164:- Systemic Gene Transfer with
73:dystrophin-associated proteins
64:, and also to proteins in the
34:dystrophin-associated proteins
1:
720:10.1001/jamaneurol.2020.1484
664:10.1016/j.bbadis.2006.06.009
162:Delandistrogene moxeparvovec
156:Therapeutic Microdystrophin
141:Duchenne muscular dystrophy
44:. The other complex is the
1517:
1428:
1218:Sliding filament mechanism
168:74.MHCK7.micro-dystrophin.
966:
937:
613:10.1186/s13023-017-0631-3
764:adisinsight.springer.com
541:Comprehensive Physiology
234:Medical Subject Headings
86:In the epithelia of the
1411:Fukutin-related protein
418:10.1242/jcs.115.14.2801
406:Journal of Cell Science
1369:Sarcoplasmic reticulum
1198:Neuromuscular junction
1106:elastic filament/titin
828:Vascular smooth muscle
460:10.1093/ptj/85.12.1372
122:striated muscle fibres
1101:thick filament/myosin
128:Clinical significance
42:striated muscle cells
553:10.1002/cphy.c140048
412:(Pt 14): 2801–2803.
308:Muscular Dystrophies
66:extracellular matrix
26:multiprotein complex
20:, also known as the
1096:thin filament/actin
1082:(a, i, and h bands;
77:sarcoglycan complex
1501:Biochemistry stubs
134:muscular dystrophy
1496:Protein complexes
1458:
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1331:Myosatellite cell
1247:Intercalated disc
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1154:Connective tissue
1072:
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1035:Synemin/desmuslin
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454:(12): 1372–1388.
366:978-0-12-803196-4
272:978-0-12-088488-9
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1451:expanding it
1443:biochemistry
1440:
1425:
1275:Muscle fiber
1045:
976:Dystrobrevin
929:Dystroglycan
864:
763:
754:
742:. Retrieved
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198:(2): 53–63.
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107:
96:
85:
81:muscle cells
70:
62:cytoskeleton
55:
45:
21:
17:
15:
1326:Muscle cell
1301:Myofilament
1120:Tropomyosin
1091:Myofilament
892:Sarcoglycan
383:|work=
289:|work=
98:Aquaporin 4
1490:Categories
1396:Telethonin
1357:Sarcolemma
1352:Sarcoplasm
1285:extrafusal
1280:intrafusal
1242:Myocardium
1203:Motor unit
1177:Endomysium
1172:Perimysium
1060:Caveolin 3
993:Syntrophin
971:Dystrophin
823:Calmodulin
744:9 November
173:References
58:dystrophin
30:dystrophin
1401:Dysferlin
1384:ungrouped
1306:Sarcomere
1292:Myofibril
1252:Nebulette
1162:Epimysium
1078:Sarcomere
1030:Dysbindin
1025:Syncoilin
942:Sarcospan
873:Membrane/
860:Costamere
606:(1): 79.
385:ignored (
375:cite book
291:ignored (
281:cite book
118:costamere
52:Structure
38:costamere
1391:Myotilin
1362:T-tubule
1322:Myoblast
1167:Fascicle
1127:Troponin
1046:related:
849:Skeletal
838:Striated
739:32539076
673:16887341
632:28446219
581:26140716
522:23097603
478:16305275
426:12082140
334:21496623
214:23738275
104:Function
92:utrophin
32:and the
1406:Fukutin
1232:Cardiac
1191:General
1111:nebulin
730:7296461
623:5405509
572:4767260
513:3476854
469:4496952
205:3666507
147:Therapy
1382:Other/
1347:Desmin
1234:muscle
851:muscle
840:muscle
815:muscle
813:Smooth
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269:
236:(MeSH)
212:
202:
166:rAAVrh
88:kidney
1441:This
1340:Other
1315:Cells
1268:Fiber
24:is a
1447:stub
1261:Both
1055:NOS1
922:SGCZ
917:SGCG
912:SGCE
907:SGCD
902:SGCB
897:SGCA
865:DAPC
746:2022
735:PMID
669:PMID
655:1772
628:PMID
577:PMID
557:ISBN
518:PMID
474:PMID
422:PMID
387:help
361:ISBN
330:PMID
320:ISBN
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267:ISBN
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