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1 l arthrogryposis-1 (DA-1), a severe skeletal muscle disorder.
2 tal muscle mass in many acquired and genetic muscle disorders.
3 veness of gene- and cell-based therapies for muscle disorders.
4 tential intracellular therapeutic target for muscle disorders.
5 omyosin, as needed in therapeutic design for muscle disorders.
6 new and personalized treatments for skeletal muscle disorders.
7 7 may be an important therapeutic target for muscle disorders.
8 tly being pursued as therapeutic options for muscle disorders.
9 promises for the treatment of many forms of muscle disorders.
10 and neuropsychiatric diseases, and skeletal muscle disorders.
11 sed this treatment strategy for pathological muscle disorders.
12 aging syndromes, lipodystrophy, and striated muscle disorders.
13 wasting in chronic diseases and degenerative muscle disorders.
14 fflicted with ischemic and nonischemic heart muscle disorders.
15 se channels are known to cause several human muscle disorders.
16 c population included patients with 12 other muscle disorders.
17 atments for muscle weakness in DMD and other muscle disorders.
18 ential therapeutic approach for degenerative muscle disorders.
19 tment of strabismus, nystagmus, or other eye muscle disorders.
20 oving regeneration in diverse and burdensome muscle disorders.
21 s in transcription factors cause a number of muscle disorders.
22 hem attractive models for studying mammalian muscle disorders.
23 ve skeletal muscle formation in degenerative muscle disorders.
24 of genetic therapy for DMD, as well as other muscle disorders.
25 the diagnosis, management, and treatment of muscle disorders.
26 ons learned from systemic genetic therapy of muscle disorders also should have implications for other
27 roderma myopathy is a heterogeneous group of muscle disorders among patients with underlying sclerode
30 everal ongoing clinical trials for different muscle disorders and the advances in the understanding o
31 were 8 subjects who were being evaluated for muscle disorders and who were not diagnosed as having ju
33 s causing FPLD, in contrast to those causing muscle disorders, are tightly clustered within the C-ter
34 auses nemaline myopathy, a lethal congenital muscle disorder associated with aberrant myofiber struct
35 -epimerase/ManNAc kinase) myopathy is a rare muscle disorder associated with aging and is related to
36 have DMD gene mutations but may have another muscle disorder associated with elevated neonatal creati
38 ic right ventricular cardiomyopathy, a heart muscle disorder associated with ventricular arrhythmias,
39 (NF-kappaB) is involved in multiple skeletal muscle disorders, but how it functions in differentiatio
40 Infantile Pompe disease is a fatal genetic muscle disorder caused by a deficiency of acid alpha-glu
42 rage myopathy (MSM) is a congenital skeletal muscle disorder caused by missense mutations in the beta
43 ongenita is a temperature-sensitive skeletal muscle disorder caused by missense mutations that occur
44 nne muscular dystrophy (DMD), a degenerative muscle disorder caused by mutations in the dystrophin ge
45 (HyperKPP) is an autosomal dominant skeletal muscle disorder caused by single mutations in the SCN4A
47 (DMD) is the most common and lethal genetic muscle disorder, caused by recessive mutations in the dy
49 ve cardiomyopathy (RCM) is an uncommon heart muscle disorder characterized by impaired filling of the
51 Inclusion body myopathy is a progressive muscle disorder characterized by nuclear and cytoplasmic
52 (OPMD) is an autosomal dominant, late-onset muscle disorder characterized by ptosis, swallowing diff
53 y myopathies (IMs) are debilitating skeletal muscle disorders characterized by common pathological ev
54 cally and genetically heterogeneous group of muscle disorders characterized by congenital or early-on
55 ibilities for the treatment of many forms of muscle disorders characterized by impaired regeneration
56 phies (MDs) comprise a group of degenerative muscle disorders characterized by progressive muscle was
58 physiologic approach to certain drug-induced muscle disorders, especially those caused by the lipid-l
60 f inflammatory myopathies and those of other muscle disorders, in particular, the genetic muscular dy
61 riteria, the most common immune inflammatory muscle disorders include Dermatomyositis (DM), Polymyosi
63 ic dystrophy (DM), the most common inherited muscle disorder, is caused by a CTG expansion in the 3"-
64 ispositioned nuclei are correlated with many muscle disorders, it is not known whether this common ph
65 otonia are three autosomal dominant skeletal muscle disorders linked to the SCN4A gene encoding the a
72 mogenic cardiomyopathy is an inherited heart muscle disorder, predisposing to sudden cardiac death, p
73 Nemaline myopathy (NM) is a rare congenital muscle disorder primarily affecting skeletal muscles tha
74 lar cardiomyopathy (ARVC) is a primary heart muscle disorder resulting from desmosomal protein mutati
75 causes such as celiac disease, thyroid, and muscle disorders should be considered in the differentia
76 Given this channel's involvement in other muscle disorders such as paramyotonia congenita and hype
78 opathy (ARVC) is an autosomal dominant heart muscle disorder that causes arrhythmia, heart failure, a
79 ic paralysis (HypoPP) is a familial skeletal muscle disorder that presents with recurrent episodes of
80 lar dystrophy is a general term encompassing muscle disorders that cause weakness and wasting, typica
81 ic paralyses are a diverse group of skeletal muscle disorders that share a common pathophysiological
82 se either a cardiac or a congenital skeletal muscle disorder through different modes of inheritance.
84 d genetically heterogeneous group of primary muscle disorders where weakness and atrophy are restrict
85 eterogeneous group of genetically determined muscle disorders with a primary or predominant involveme
87 comprise a heterogeneous group of heritable muscle disorders with often difficult to interpret muscl
88 stem cell-based therapies to treat skeletal muscle disorders, with a special emphasis on muscular dy
89 NaV1.4 give rise to a heterogeneous group of muscle disorders, with gain-of-function defects causing
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