ライフサイエンスコーパス: muscular dystrophy

1 fter injury and in a mouse model of Duchenne muscular dystrophy.

2 model for ambulatory-type Lmalpha2-deficient muscular dystrophy.

3 lar dystrophy, including the lethal Duchenne muscular dystrophy.

4 trophic mdx mice, a murine model of Duchenne muscular dystrophy.

5 agonist SR8278 could slow the progression of muscular dystrophy.

6 mutations in SMCHD1 contribute to a type of muscular dystrophy.

7 Thbs4 as a potential therapeutic target for muscular dystrophy.

8 ystrophic symptoms in this model of Duchenne muscular dystrophy.

9 d and likely contributes to the pathology of muscular dystrophy.

10 ic muscles in mdx mice, a model for Duchenne muscular dystrophy.

11 terstitial cells (PICs), play a dual role in muscular dystrophy.

12 reframing, similar to observations in Becker muscular dystrophy.

13 ogy, and a functional deficit reminiscent of muscular dystrophy.

14 tiveness of potential therapies for Duchenne muscular dystrophy.

15 as a key event in the aetiology of Duchenne muscular dystrophy.

16 nd cellular attachment, which often leads to muscular dystrophy.

17 cells robustly engraft into a mouse model of muscular dystrophy.

18 thology in patients with facioscapulohumeral muscular dystrophy.

19 t and may provide an effective treatment for muscular dystrophy.

20 peutic targets for the treatment of Duchenne muscular dystrophy.

21 of mdx(5cv) mice, a mouse model for Duchenne muscular dystrophy.

22 d with many diseases, including ischemia and muscular dystrophy.

23 le mouse models of human diseases, including muscular dystrophy.

24 lker-Warburg syndrome to Fukuyama congenital muscular dystrophy.

25 of the two drugs for fukutin-related protein-muscular dystrophy.

26 ce of the protein dystrophin causes Duchenne muscular dystrophy.

27 mbrane resealing; loss of dysferlin leads to muscular dystrophy.

28 nd utrophin-binding protein, ameliorates mdx muscular dystrophy.

29 dystrophin gene mutations who have Duchenne muscular dystrophy.

30 dystrophin gene, is the most common form of muscular dystrophy.

31 treatment option for patients with Duchenne muscular dystrophy.

32 normally without signs of skin blistering or muscular dystrophy.

33 e BM structure, and substantially ameliorate muscular dystrophy.

34 and underlies a recessive form of inherited muscular dystrophy.

35 elopment as a therapy for Duchenne or Becker muscular dystrophy.

36 espan and survival in patients with Duchenne muscular dystrophy.

37 ed dystrophin restoration in mouse models of muscular dystrophy.

38 ass in models of osteogenesis imperfecta and muscular dystrophy.

39 nsferase 1 (POMT1) and POMT2 underlie severe muscular dystrophies.

40 TMTC3 COB phenotype from typical congenital muscular dystrophies.

41 ex, is at the center of molecular studies of muscular dystrophies.

42 cle and is similarly affected in many of the muscular dystrophies.

43 reatment of degenerative disorders including muscular dystrophies.

44 ex (DAPC), both of which have been linked to muscular dystrophies.

45 asis and in pathogenesis of diseases such as muscular dystrophies.

46 encoding genes cause congenital/limb-girdle muscular dystrophies.

47 on susceptibility to obesity, diabetes, and muscular dystrophies.

48 alpha-dystroglycan gives rise to congenital muscular dystrophies.

49 Fibrosis is the main complication of muscular dystrophies.

50 omechanisms of neurological abnormalities in muscular dystrophies.

51 s of Duchenne muscular dystrophy, congenital muscular dystrophy 1A, and limb girdle muscular dystroph

52 lar dystrophy 2I (LGMD2I), severe congenital muscular dystrophy 1C (MDC1C), to Walker-Warburg Syndrom

53 Limb girdle muscular dystrophy 2A is due to loss-of-function mutatio

54 ember calpain 3 (CAPN3) underlie limb-girdle muscular dystrophy 2A.

55 ysferlin, the protein missing in limb girdle muscular dystrophy 2B and Miyoshi myopathy, concentrates

56 in dysferlin are responsible for limb girdle muscular dystrophy 2B and Miyoshi myopathy.

57 ions in patients presenting with limb-girdle muscular dystrophy 2B.

58 nital muscular dystrophy 1A, and limb girdle muscular dystrophy 2D.

59 Limb girdle muscular dystrophy 2H is caused by mutations in the gene

60 e range of pathologies from mild limb girdle muscular dystrophy 2I (LGMD2I), severe congenital muscul

61 tant mouse representing moderate limb-girdle muscular dystrophy 2I.

62 cal phenotypes, most commonly as limb-girdle muscular dystrophy 2I.

63 RP P448Lneo(-) mice, a model for limb girdle muscular dystrophy 2I.

64 oas sparing is common in facioscapulohumeral muscular dystrophy (67% of the patients).

65 These studies identify a novel mechanism of muscular dystrophy: a blunted transcriptional response t

66 in a laminin-alpha2 knockout mouse model of muscular dystrophy, acting as a link between alpha-DG an

67 myoblasts involved in the pathophysiology of muscular dystrophies and confirmed our results in vivo b

68 In models of muscular dystrophy and cancer cachexia, combined inhibit

69 Mutations in its gene have been linked to muscular dystrophy and cardiomyopathy.

70 was first discovered as a candidate gene for muscular dystrophy and cardiomyopathy.

71 n mouse models of acute muscle injury and in muscular dystrophy and determined that both regimens pro

72 Cells derived from patients with muscular dystrophy and differentiated using this protoco

73 ults for non-DMD disorders, including Becker muscular dystrophy and forms of limb-girdle and congenit

74 modifying a pathologically distinct form of muscular dystrophy and increased the production of a tru

75 tance of neutrophil-mediated inflammation in muscular dystrophy and indicate elastase-mediated regula

76 tory role of COUP-TFII in the development of muscular dystrophy and open up a potential therapeutic o

77 However, for Duchenne muscular dystrophy and other rare diseases, these requir

78 open up new therapeutic avenues for Duchenne muscular dystrophy and possibly other neuromuscular dise

79 te of pharmacological therapies for Duchenne muscular dystrophy and related disorders.

80 velopment of SSOs designed to treat Duchenne muscular dystrophy and spinal muscular atrophy, which ar

81 ophy and forms of limb-girdle and congenital muscular dystrophies, and 21 patients had false-negative

82 echnology to treat mouse models of diabetes, muscular dystrophy, and acute kidney disease.

83 ular disorders including Huntington disease, muscular dystrophy, and amyotrophic lateral sclerosis.

84 (POMT2) are known to cause severe congenital muscular dystrophy, and recently, mutations in POMT2 hav

85 ons, cerebral palsy, dilated cardiomyopathy, muscular dystrophy, and schizophrenia.

86 Limb-girdle muscular dystrophies are a genetically diverse group of

87 Muscular dystrophies are characterized by weakness and w

88 The muscular dystrophies are genetically diverse.

89 Congenital muscular dystrophies are hereditary disorders characteri

90 linked to several of the Lmalpha2-deficient muscular dystrophies are predicted to compromise polymer

91 tive effects of chronic steroid treatment in muscular dystrophy are paradoxical because these steroid

92 a model that configures facioscapulohumeral muscular dystrophy as a "muscle-by-muscle" disease.

93 s dystroglycanopathies, which are congenital muscular dystrophies associated with brain and eye anoma

94 Mutations in POMTs cause severe muscular dystrophies associated with pronounced neurolog

95 male patients aged 2-28 years with Duchenne muscular dystrophy at 20 centres in nine countries.

96 Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD), interventions reducing the pro

97 Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD), X-linked dilated cardiomyopath

98 rved in calf muscles of patients with Becker muscular dystrophy (BMD, n = 14) and limb-girdle type 2I

99 loss of ambulation in patients with Duchenne muscular dystrophy but are accompanied by prominent adve

100 is a strategy for the treatment of Duchenne muscular dystrophy, but has variable efficacy.

101 sults, PDGF-BB may play a protective role in muscular dystrophies by enhancing muscle regeneration th

102 ver, the impact of this approach on Duchenne muscular dystrophy cardiac function has yet to be evalua

103 re linked to approximately 30% of congenital muscular dystrophy cases.

104 ystrophy (MDC1A) is a devastating congenital muscular dystrophy caused by mutations in the LAMA2 gene

105 ular dystrophy is a severe inherited form of muscular dystrophy caused by mutations in the reading fr

106 n and severe form among children is Duchenne muscular dystrophy, caused by mutations in the dystrophi

107 alpha-Dystroglycanopathies are a group of muscular dystrophies characterized by alpha-DG hypoglyco

108 elated dystroglycanopathies, another form of muscular dystrophy characterized by weak interactions be

109 nt DMD cohorts: cohort 1 (The Parent Project Muscular Dystrophy-Cincinnati Children's Hospital Medica

110 muscle pathology in mouse models of Duchenne muscular dystrophy, congenital muscular dystrophy 1A, an

111 Thus, epigenetic silencing of klotho during muscular dystrophy contributes substantially to lost reg

112 and POMT2, underlie a subgroup of congenital muscular dystrophies designated alpha-dystroglycanopathi

113 Congenital muscular dystrophies display a wide phenotypic and genet

114 of mice representing Duchenne and congenital muscular dystrophies (DMD and CMD, respectively) and dys

115 Dystrophinopathies include Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (

116 In Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (

117 muscle ultrasound data in boys with Duchenne muscular dystrophy (DMD) and healthy controls to determi

118 eletion or duplication genotypes of Duchenne muscular dystrophy (DMD) due to different fluorescent in

119 novel therapeutics for treatment of Duchenne muscular dystrophy (DMD) has led to clinical trials that

120 Duchenne muscular dystrophy (DMD) impacts 1 : 3500 boys and leads

121 Duchenne muscular dystrophy (DMD) is a candidate for the recommen

122 Duchenne muscular dystrophy (DMD) is a debilitating X-linked diso

123 Duchenne muscular dystrophy (DMD) is a devastating disease affect

124 Duchenne muscular dystrophy (DMD) is a fatal X-linked disorder ca

125 Duchenne muscular dystrophy (DMD) is a genetic disease characteri

126 Duchenne muscular dystrophy (DMD) is a genetic disorder that caus

127 Duchenne muscular dystrophy (DMD) is a genetic neuromuscular diso

128 Duchenne muscular dystrophy (DMD) is a lethal muscle disease invo

129 Duchenne muscular dystrophy (DMD) is a muscular dystrophy with hi

130 Duchenne muscular dystrophy (DMD) is a neuromuscular disease that

131 Duchenne muscular dystrophy (DMD) is a severe and progressive mus

132 Duchenne Muscular Dystrophy (DMD) is a severe muscle disorder cau

133 Duchenne muscular dystrophy (DMD) is a severe, degenerative muscl

134 Duchenne muscular dystrophy (DMD) is a severe, degenerative muscl

135 Duchenne muscular dystrophy (DMD) is a severe, progressive, and r

136 Duchenne muscular dystrophy (DMD) is an incurable X-linked geneti

137 Duchenne muscular dystrophy (DMD) is an incurable X-linked muscle

138 Duchenne muscular dystrophy (DMD) is an X-linked disorder with dy

139 Duchenne muscular dystrophy (DMD) is an X-linked progressive dege

140 Duchenne muscular dystrophy (DMD) is an X-linked recessive diseas

141 Duchenne Muscular Dystrophy (DMD) is caused by a lack of dystroph

142 Duchenne muscular dystrophy (DMD) is caused by dystrophin deficie

143 Duchenne muscular dystrophy (DMD) is characterized by a progressi

144 Duchenne muscular dystrophy (DMD) is characterized by muscle dege

145 enhances exon-skipping activity in Duchenne muscular dystrophy (DMD) mdx mice.

146 proves skeletal muscle pathology in Duchenne muscular dystrophy (DMD) mouse models.

147 bnormally elevated in the muscle of Duchenne muscular dystrophy (DMD) patients and animal models.

148 are highly enriched in the serum of Duchenne Muscular Dystrophy (DMD) patients and dystrophic mouse m

149 a standard palliative treatment for Duchenne muscular dystrophy (DMD) patients, but various adverse e

150 ) are abnormally present in sera of Duchenne muscular dystrophy (DMD) patients, limb-girdle muscular

151 ly tested the implication of ApN in Duchenne muscular dystrophy (DMD) using mdx mice, a model of DMD,

152 urrently no effective treatment for Duchenne muscular dystrophy (DMD), a lethal monogenic disorder ca

153 Duchenne muscular dystrophy (DMD), caused by mutations at the dys

154 promising therapeutic strategy for Duchenne muscular dystrophy (DMD), employing morpholino antisense

155 nt from the pathogenic mutation: in Duchenne muscular dystrophy (DMD), for instance, age at loss of a

156 gence of experimental therapies for Duchenne muscular dystrophy (DMD), it is fundamental to understan

157 In Duchenne muscular dystrophy (DMD), loss of dystrophin leads to th

158 ngue weakness, like all weakness in Duchenne muscular dystrophy (DMD), occurs as a result of contract

159 scle histopathology associated with Duchenne Muscular Dystrophy (DMD), the molecular and cellular mec

160 treating genetic diseases, such as Duchenne muscular dystrophy (DMD), which is caused by mutations i

161 y are needed for clinical trials in Duchenne muscular dystrophy (DMD).

162 ors hold great promise for treating Duchenne muscular dystrophy (DMD).

163 y and drive muscle deterioration in Duchenne muscular dystrophy (DMD).

164 Its absence leads to Duchenne muscular dystrophy (DMD).

165 rophin, a known disease modifier in Duchenne muscular dystrophy (DMD).

166 is still no curative treatment for Duchenne muscular dystrophy (DMD).

167 in are a possible means of treating Duchenne Muscular Dystrophy (DMD).

168 t dystrophin translation, and cause Duchenne muscular dystrophy (DMD).

169 reclinical models and subjects with Duchenne muscular dystrophy (DMD).

170 r contributors to muscle wasting in Duchenne muscular dystrophy (DMD).

171 of mdx mice (i.e., a mouse model of Duchenne Muscular Dystrophy [DMD]) could restore the morphology o

172 phic mesoangioblasts from a Golden Retriever muscular dystrophy dog were transfected with the large-s

173 Analysis of four additional GMPPB-associated muscular dystrophy dystroglycanopathy cases by electromy

174 In preclinical models for Duchenne muscular dystrophy, dystrophin restoration during adeno-

175 In individuals exhibiting congenital muscular dystrophy, early-onset cataracts, and mild inte

176 expression of genes linked to Emery-Dreifuss muscular dystrophy (EDMD) and centronuclear myopathy (CN

177 ients with autosomal dominant Emery-Dreifuss muscular dystrophy (EDMD) as well as dilated cardiomyopa

178 dipogenic progenitors in facioscapulohumeral muscular dystrophy.Facioscapulohumeral muscular dystroph

179 hosphonates have been used to treat Duchenne muscular dystrophy for prevention of osteoporosis.

180 rophy type 2A is the most common limb girdle muscular dystrophy form worldwide.

181 teins and its dysfunction leads to a form of muscular dystrophy frequently associated with neurodevel

182 used for distinguishing facioscapulohumeral muscular dystrophy from other myopathies in selected cas

183 Facioscapulohumeral muscular dystrophy (FSHD) involves sporadic expression o

184 Facioscapulohumeral muscular dystrophy (FSHD) is a prevalent, incurable myop

185 Facioscapulohumeral muscular dystrophy (FSHD) is an autosomal dominant neuro

186 ave been associated with facioscapulohumeral muscular dystrophy (FSHD) type 2.

187 with the pathogenesis of Facioscapulohumeral muscular dystrophy (FSHD).

188 ption factor DUX4 causes facioscapulohumeral muscular dystrophy (FSHD).

189 ociated genetic disease, facioscapulohumeral muscular dystrophy (FSHD1A, MIM 158900).

190 INTERPRETATION: In patients with Duchenne muscular dystrophy, glucocorticoid treatment is associat

191 ed on a total of 12 treated golden retriever muscular dystrophy (GRMD) dogs.

192 of ANO5 myopathies with dysferlin-associated muscular dystrophies has inspired the hypothesis that AN

193 a point mutation in Dmd)-a model of Duchenne muscular dystrophy-Hippo deficiency protected against ov

194 ecommended as a standard of care in Duchenne muscular dystrophy; however, few studies have assessed t

195 and V428D) that result in severe congenital muscular dystrophies in humans, in yeast Pmt4 (I112R and

196 activity as a contributing factor underlying muscular dystrophy in a dystrophin-deficient murine mode

197 uman patient that defects in TMEM5 result in muscular dystrophy in combination with abnormal brain de

198 vailable therapy exists to potentially treat muscular dystrophy in DM1.

199 cy does not provide sustained improvement of muscular dystrophy in mdx(5cv) mice.

200 opathy, giving rise to a more severe form of muscular dystrophy in the Dysf(B6) mouse model through i

201 Shared pathological features among muscular dystrophies include breakdown, or loss of muscl

202 of steroid administration in other types of muscular dystrophies, including limb-girdle muscular dys

203 DGC deficiency in humans results in muscular dystrophy, including the lethal Duchenne muscul

204 operative effort of stakeholders in Duchenne muscular dystrophy-including representatives from patien

205 In the degenerative disease Duchenne muscular dystrophy, inflammatory cells enter muscles in

206 Hence, Ctss induction during muscular dystrophy is a pathologic event that partially

207 Duchenne muscular dystrophy is a rare, progressive, muscle-wastin

208 Duchenne muscular dystrophy is a severe and progressive striated

209 RATIONALE: Duchenne muscular dystrophy is a severe inherited form of muscula

210 meral muscular dystrophy.Facioscapulohumeral muscular dystrophy is a severe myopathy that is caused b

211 Facioscapulohumeral muscular dystrophy is a slowly progressive but devastati

212 Fukutin-related protein-muscular dystrophy is characterized by defects in glycos

213 Muscular dystrophy is characterized by progressive skele

214 proaches to restore dystrophin expression in muscular dystrophy is obtaining a sufficient quantity of

215 hies type 2I (LGMD2I), a recessive autosomal muscular dystrophy, is caused by mutations in the Fukuti

216 ment of fibrosis and chronic inflammation in muscular dystrophy, less is known about how they are mec

217 ave also been linked to a milder limb-girdle muscular dystrophy (LGMD) phenotype, named LGMD type 2N

218 clusion body myopathy (hIBM) and limb-girdle muscular dystrophy (LGMD), are a genetically heterogeneo

219 of 4 with cardiac arrhythmia and limb-girdle muscular dystrophy (LGMD).

220 h sarcoglycanopathies, which are limb-girdle muscular dystrophies (LGMD2C-2F) caused by mutations in

221 trophy (BMD, n = 14) and limb-girdle type 2I muscular dystrophy (LGMD2I, n = 11), before and after co

222 group of MD's referred to as the limb-girdle muscular dystrophies (LGMDs) can affect boys or girls, w

223 muscular dystrophies, including limb-girdle muscular dystrophies (LGMDs).

224 imprinted genes, and the facioscapulohumeral muscular dystrophy locus.

225 als and developing refined therapies for the muscular dystrophies (MD's) and other myogenic disorders

226 Muscular dystrophy (MD) is associated with mutations in

227 Lama2-related muscular dystrophy (MDC1A) is a devastating congenital m

228 Muscular dystrophies (MDs) are often characterized by im

229 uilds muscle and bone was tested in X-linked muscular dystrophy mice (mdx).

230 hesized that a reduction of Collagen VI in a muscular dystrophy model that presents with fibrosis wou

231 ury and reduced myofiber size decline in the muscular dystrophy model.

232 is finding to develop an facioscapulohumeral muscular dystrophy mouse model with muscle-specific doxy

233 muscle atrophy and dysfunction in a Duchenne muscular dystrophy mouse model.

234 tal muscles in addition to heart in Duchenne muscular dystrophy mouse models and that mineralocortico

235 d in skeletal muscle of classical congenital muscular dystrophy mouse models.

236 notype in the mdx (a mouse model of Duchenne muscular dystrophy) mouse by blunting the regeneration o

237 letal muscle regenerates, but with age or in muscular dystrophies, muscle is replaced by fat.

238 function is compromised, such as myopathies, muscular dystrophies, neuromuscular diseases, and age-re

239 Oculopharyngeal muscular dystrophy (OPMD) is a late onset disease caused

240 Oculopharyngeal muscular dystrophy (OPMD) is an autosomal dominant, late

241 ered in the transcriptome of oculopharyngeal muscular dystrophy (OPMD).

242 ar-1 (PABPN1) protein causes oculopharyngeal muscular dystrophy (OPMD).

243 of muscular dystrophy termed oculopharyngeal muscular dystrophy (OPMD).

244 ns of Tmem2 and the etiologies of congenital muscular dystrophies, particularly dystroglycanopathies.

245 tic background, resulting in protection from muscular dystrophy pathogenesis that included reduced my

246 o the known contribution of myeloid cells to muscular dystrophy pathology.

247 at this mechanism is disrupted in congenital muscular dystrophy patient myotubes carrying a nonsense

248 Our data align the D2-mdx with Duchenne muscular dystrophy patients with the LTBP4 genetic modif

249 athy is a leading cause of death in Duchenne muscular dystrophy patients, and currently no effective

250 integrin observed in mdx muscle and Duchenne muscular dystrophy patients.

251 ly, CD82 expression is decreased in Duchenne muscular dystrophy patients.

252 s progressive muscle pathology resembles the muscular dystrophy phenotype in humans and mice lacking

253 the DBA/2J background confers a more severe muscular dystrophy phenotype than the original strain, d

254 nerating myofibers of patients with Duchenne muscular dystrophy, polymyositis, and compartment syndro

255 Parent Project Muscular Dystrophy (PPMD) convened a workshop in Bethesd

256 In Duchenne muscular dystrophy, progressive loss of muscle tissue is

257 overlap were due to mutations in congenital muscular dystrophy-related genes (4 families) and collag

258 Muscular dystrophies result from a defect in the linkage

259 Dysferlinopathies are a group of muscular dystrophies resulting from a genetic deficiency

260 has shown great clinical promise in Duchenne muscular dystrophy, resulting in the production of dystr

261 date the etiology of neurological defects in muscular dystrophies.SIGNIFICANCE STATEMENT Protein O-ma

262 show that the endogenous facioscapulohumeral muscular dystrophy-specific DUX4 polyadenylation signal

263 CRISPR-Cas9, neuromuscular disease, Duchenne muscular dystrophy, spinal muscular atrophy, amyotrophic

264 g neuromuscular diseases, including Duchenne muscular dystrophy, spinal muscular atrophy, amyotrophic

265 ding neuromuscular diseases such as Duchenne muscular dystrophy, spinal muscular atrophy, amyotrophic

266 onal programs, transition to adult care; (3) muscular dystrophy, spinal muscular atrophy, cystic fibr

267 utic purposes; and we will review its use in muscular dystrophy studies where considerable progress h

268 ds affects muscle remodeling in non-Duchenne muscular dystrophies, suggesting a positive outcome asso

269 that mutations in INPP5K cause a congenital muscular dystrophy syndrome with short stature, cataract

270 NA binding protein causes a specific form of muscular dystrophy termed oculopharyngeal muscular dystr

271 tein dystroglycan cause a form of congenital muscular dystrophy that is frequently associated with ne

272 ected development of treatments for Duchenne muscular dystrophy; this approach could serve as a parad

273 l muscle integrity and its susceptibility to muscular dystrophy through organization of membrane atta

274 These data link congenital muscular dystrophies to defective phosphoinositide 5-pho

275 Limb Girdle Muscular Dystrophies type 2I (LGMD2I), a recessive autos

276 Merosin-deficient congenital muscular dystrophy type 1A (MDC1A) is a dramatic neuromu

277 Limb-girdle muscular dystrophy type 1D (LGMD1D) is caused by dominan

278 SMCHD1 mutations cause facioscapulohumeral muscular dystrophy type 2 (FSHD2) via a trans-acting los

279 Limb girdle muscular dystrophy type 2A is the most common limb girdl

280 nts deficient in calpain 3 as in limb girdle muscular dystrophy type 2A, albeit in a milder form in m

281 enerally milder than observed in limb girdle muscular dystrophy type 2A, but affected the same muscle

282 arcoglycan-null mice, a model of limb-girdle muscular dystrophy type 2C, with a Col6a2-deficient mous

283 scular dystrophy (DMD) patients, limb-girdle muscular dystrophy type 2D (LGMD2D) and their respective

284 al clinical phenotypes including limb-girdle muscular dystrophy type 2L and Miyoshi myopathy type 3,

285 Congenital muscular dystrophy type MDC1A is caused by mutations in

286 s to treat genetic diseases such as Duchenne muscular dystrophy, we propose that exon skipping of Fce

287 n responsible for the majority of congenital muscular dystrophies when dysfunctional, has a function

288 r chronic muscle conditions such as Duchenne muscular dystrophy, where their use is associated with p

289 (DM1) is the most common form of adult-onset muscular dystrophy, which is characterised by progressiv

290 l malformation and reproductive disorders to muscular dystrophy, which we speculate to be consistent

291 and accelerates disease in 2 mouse models of muscular dystrophy, while overexpression of mouse Thbs4

292 al trial of patients with Duchenne or Becker muscular dystrophy whose LVEF was preserved and MF was p

293 he standard treatment for Duchenne and other muscular dystrophies with serious adverse effects, inclu

294 ng with variable clinical features including muscular dystrophy with a reduction in dystroglycan glyc

295 or mouse Thbs4 rescues a Drosophila model of muscular dystrophy with augmented membrane residence of

296 background therapy in patients with Duchenne muscular dystrophy with early myocardial disease.

297 Duchenne muscular dystrophy (DMD) is a muscular dystrophy with high incidence of learning and b

298 isms that lead to intellectual disability in muscular dystrophies without associated brain malformati

299 positive fibers in a mouse model of Duchenne muscular dystrophy without apparent toxicity.

300 niversal small molecule therapy for Duchenne muscular dystrophy would be an enormous advance for this