ライフサイエンスコーパス: Duchenne muscular dystrophy

1 s, both after injury and in a mouse model of Duchenne muscular dystrophy.

2 in muscular dystrophy, including the lethal Duchenne muscular dystrophy.

3 eat the dystrophic symptoms in this model of Duchenne muscular dystrophy.

4 dystrophic muscles in mdx mice, a model for Duchenne muscular dystrophy.

5 the effectiveness of potential therapies for Duchenne muscular dystrophy.

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

7 as therapeutic targets for the treatment of Duchenne muscular dystrophy.

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

9 Absence of the protein dystrophin causes Duchenne muscular dystrophy.

10 mans with dystrophin gene mutations who have Duchenne muscular dystrophy.

11 nts a new treatment option for patients with Duchenne muscular dystrophy.

12 VL; ie, high-force contractions) accelerates Duchenne muscular dystrophy.

13 on 5 of the Acvr2b gene, in a mouse model of Duchenne muscular dystrophy.

14 protective therapy on event-free survival in Duchenne muscular dystrophy.

15 ory failure is the leading cause of death in Duchenne muscular dystrophy.

16 mitigated muscle damage in a murine model of Duchenne muscular dystrophy.

17 epresent a promising therapeutic approach in Duchenne muscular dystrophy.

18 skeletal and cardiac muscle degeneration in Duchenne muscular dystrophy.

19 ellite cell-like progenitors and a model for Duchenne Muscular Dystrophy.

20 dels of congenital muscular dystrophy 1A and Duchenne muscular dystrophy.

21 nse investigation as a potential therapy for Duchenne muscular dystrophy.

22 se loss of dystrophin expression and lead to Duchenne muscular dystrophy.

23 ogical relevance in an experimental model of Duchenne muscular dystrophy.

24 ytoskeletal protein, absence of which causes Duchenne muscular dystrophy.

25 in testing the efficacy of therapeutics for Duchenne muscular dystrophy.

26 ement of muscle function in a mouse model of Duchenne muscular dystrophy.

27 insight into cardiomyopathy associated with Duchenne muscular dystrophy.

28 efective nNOS homeostasis in dystrophin-null Duchenne muscular dystrophy.

29 y of the experimental studies are focused on Duchenne muscular dystrophy.

30 eneration occurring in a C. elegans model of Duchenne muscular dystrophy.

31 to the pathology of muscle diseases such as Duchenne muscular dystrophy.

32 d indicate that miR-206 slows progression of Duchenne muscular dystrophy.

33 linical trials for genetic disorders such as Duchenne muscular dystrophy.

34 her rare diseases, including haemophilia and Duchenne muscular dystrophy.

35 the most commonly used preclinical model for Duchenne muscular dystrophy.

36 lity of the clamp method in the mdx model of Duchenne muscular dystrophy.

37 t exons commonly deleted in individuals with Duchenne muscular dystrophy.

38 rapy are lacking for muscle diseases such as Duchenne muscular dystrophy.

39 t (dKO-Hom) mouse is a murine model of human Duchenne muscular dystrophy.

40 molecules have been identified for treating Duchenne muscular dystrophy.

41 sis of a dystrophin-deficient mouse model of Duchenne muscular dystrophy.

42 es in dystrophic mdx mice, a murine model of Duchenne muscular dystrophy.

43 s the lifespan and survival in patients with Duchenne muscular dystrophy.

44 s of genome editing in preclinical models of Duchenne muscular dystrophy(1-6), however, the long-term

45 Duchenne muscular dystrophy, a fatal degenerative muscle

46 In the most common form, Duchenne muscular dystrophy, a few personalised therapie

47 EOMs are uniquely spared in Duchenne muscular dystrophy and animal models of dystrop

48 re present in muscle lesions associated with Duchenne muscular dystrophy and dystrophin-deficient mdx

49 is a leading cause of death in patients with Duchenne muscular dystrophy and myocardial damage preced

50 most common form of muscular dystrophy after Duchenne muscular dystrophy and one of the most common a

51 However, for Duchenne muscular dystrophy and other rare diseases, the

52 use, may open up new therapeutic avenues for Duchenne muscular dystrophy and possibly other neuromusc

53 In boys with Duchenne muscular dystrophy and preserved ejection fract

54 ong a suite of pharmacological therapies for Duchenne muscular dystrophy and related disorders.

55 ht the development of SSOs designed to treat Duchenne muscular dystrophy and spinal muscular atrophy,

56 This success, for Duchenne muscular dystrophy and spinal muscular atrophy,

57 k for providing information to patients with Duchenne muscular dystrophy and their families when intr

58 ts in this young population with early-stage Duchenne muscular dystrophy are encouraging but need to

59 rs of the inherited muscle wasting condition Duchenne muscular dystrophy, as they allow non-invasive

60 r (LV) dysfunction before the onset of overt Duchenne muscular dystrophy-associated cardiomyopathy (D

61 enrolled male patients aged 2-28 years with Duchenne muscular dystrophy at 20 centres in nine countr

62 to delay loss of ambulation in patients with Duchenne muscular dystrophy but are accompanied by promi

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

64 However, the impact of this approach on Duchenne muscular dystrophy cardiac function has yet to

65 ost common and severe form among children is Duchenne muscular dystrophy, caused by mutations in the

66 normally increased in the mdx mouse model of Duchenne muscular dystrophy compared with the wild-type

67 pment of muscle pathology in mouse models of Duchenne muscular dystrophy, congenital muscular dystrop

68 oss underlying contraction-induced injury in Duchenne muscular dystrophy distinctly different from th

69 Duchenne muscular dystrophy (DMD) affects 1 in 3500 live

70 Dystrophinopathies include Duchenne muscular dystrophy (DMD) and Becker muscular dy

71 In Duchenne muscular dystrophy (DMD) and Becker muscular dy

72 titative muscle ultrasound data in boys with Duchenne muscular dystrophy (DMD) and healthy controls t

73 dystrophy has been evaluated in humans with Duchenne Muscular Dystrophy (DMD) and in mouse models in

74 th multiple inflammatory states, severity of Duchenne muscular dystrophy (DMD) and muscle size in hea

75 ve involvement of upper extremity muscles in Duchenne muscular dystrophy (DMD) and showed the feasibi

76 alization contributes to the pathogenesis of Duchenne muscular dystrophy (DMD) by promoting functiona

77 Treatment of Duchenne muscular dystrophy (DMD) by replacing mutant dy

78 Duchenne muscular dystrophy (DMD) causes severe disabili

79 asingly considered to be outcome measures in Duchenne muscular dystrophy (DMD) clinical trials.

80 at loss of ambulation (LoA) in a multiethnic Duchenne muscular dystrophy (DMD) cohort.

81 Patients affected by Duchenne muscular dystrophy (DMD) develop a progressive

82 Duchenne muscular dystrophy (DMD) displays a clinical ra

83 ly reported attempt was performed in newborn Duchenne muscular dystrophy (DMD) dogs.

84 ish the deletion or duplication genotypes of Duchenne muscular dystrophy (DMD) due to different fluor

85 The essential product of the Duchenne muscular dystrophy (DMD) gene is dystrophin(1),

86 pment of novel therapeutics for treatment of Duchenne muscular dystrophy (DMD) has led to clinical tr

87 Duchenne muscular dystrophy (DMD) impacts 1 : 3500 boys

88 r the efficacy of experimental therapies for Duchenne Muscular Dystrophy (DMD) in clinical trials.

89 Duchenne muscular dystrophy (DMD) induces sarcolemmal me

90 Exon-skipping therapies aim to convert Duchenne muscular dystrophy (DMD) into less severe Becke

91 Duchenne muscular dystrophy (DMD) is a candidate for the

92 Duchenne muscular dystrophy (DMD) is a classical monogen

93 Duchenne muscular dystrophy (DMD) is a common and relent

94 Duchenne muscular dystrophy (DMD) is a debilitating X-li

95 Duchenne muscular dystrophy (DMD) is a degenerative skel

96 Duchenne muscular dystrophy (DMD) is a devastating disea

97 Duchenne muscular dystrophy (DMD) is a devastating muscl

98 Duchenne muscular dystrophy (DMD) is a devastating neuro

99 Duchenne muscular dystrophy (DMD) is a devastating progr

100 Duchenne muscular dystrophy (DMD) is a devastating X-lin

101 Duchenne muscular dystrophy (DMD) is a fatal degenerativ

102 Duchenne muscular dystrophy (DMD) is a fatal genetic dis

103 Duchenne muscular dystrophy (DMD) is a fatal muscle diso

104 Duchenne muscular dystrophy (DMD) is a fatal muscle-wast

105 Duchenne muscular dystrophy (DMD) is a fatal neuromuscul

106 Duchenne muscular dystrophy (DMD) is a fatal neuromuscul

107 Duchenne muscular dystrophy (DMD) is a fatal X-linked di

108 Duchenne muscular dystrophy (DMD) is a fatal X-linked di

109 Duchenne muscular dystrophy (DMD) is a fatal, X-linked n

110 Duchenne muscular dystrophy (DMD) is a genetic disease c

111 Duchenne muscular dystrophy (DMD) is a genetic disorder

112 Duchenne muscular dystrophy (DMD) is a genetic disorder

113 Duchenne muscular dystrophy (DMD) is a genetic neuromusc

114 Duchenne muscular dystrophy (DMD) is a lethal genetic di

115 Duchenne muscular dystrophy (DMD) is a lethal muscle dis

116 Duchenne Muscular Dystrophy (DMD) is a lethal muscle dis

117 Duchenne muscular dystrophy (DMD) is a lethal neuromuscu

118 Duchenne muscular dystrophy (DMD) is a lethal, degenerat

119 Duchenne muscular dystrophy (DMD) is a lethal, muscle de

120 Duchenne muscular dystrophy (DMD) is a lethal, X-linked

121 Duchenne muscular dystrophy (DMD) is a lethal, X-linked

122 Duchenne muscular dystrophy (DMD) is a monogenic disorde

123 Duchenne muscular dystrophy (DMD) is a muscular dystroph

124 Duchenne muscular dystrophy (DMD) is a neuromuscular dis

125 Duchenne muscular dystrophy (DMD) is a progressive muscl

126 Duchenne muscular dystrophy (DMD) is a progressive neuro

127 Duchenne muscular dystrophy (DMD) is a rare genetic dise

128 Duchenne muscular dystrophy (DMD) is a severe and progre

129 Duchenne muscular dystrophy (DMD) is a severe disorder c

130 Duchenne Muscular Dystrophy (DMD) is a severe muscle dis

131 Duchenne muscular dystrophy (DMD) is a severe X-linked n

132 Duchenne muscular dystrophy (DMD) is a severe, degenerat

133 Duchenne muscular dystrophy (DMD) is a severe, degenerat

134 Duchenne muscular dystrophy (DMD) is a severe, progressi

135 Duchenne muscular dystrophy (DMD) is a uniformly fatal c

136 The cardiomyopathy of Duchenne muscular dystrophy (DMD) is an important cause

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

138 Duchenne muscular dystrophy (DMD) is an incurable X-link

139 Duchenne muscular dystrophy (DMD) is an inherited X-link

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

141 Duchenne muscular dystrophy (DMD) is an X-linked genetic

142 Duchenne muscular dystrophy (DMD) is an X-linked progres

143 Duchenne muscular dystrophy (DMD) is an X-linked recessi

144 Duchenne muscular dystrophy (DMD) is an X-linked, lethal

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

146 Duchenne muscular dystrophy (DMD) is caused by a lack of

147 Duchenne muscular dystrophy (DMD) is caused by absence o

148 Duchenne muscular dystrophy (DMD) is caused by an X-link

149 Duchenne muscular dystrophy (DMD) is caused by dystrophi

150 Duchenne muscular dystrophy (DMD) is caused by loss of d

151 Duchenne muscular dystrophy (DMD) is caused by loss of d

152 Duchenne muscular dystrophy (DMD) is caused by mutations

153 Duchenne muscular dystrophy (DMD) is caused by mutations

154 Duchenne muscular dystrophy (DMD) is caused by mutations

155 Duchenne muscular dystrophy (DMD) is characterised by pr

156 Duchenne muscular dystrophy (DMD) is characterized by a

157 Duchenne muscular dystrophy (DMD) is characterized by mu

158 Duchenne muscular dystrophy (DMD) is characterized by pr

159 Duchenne muscular dystrophy (DMD) is characterized by pr

160 Duchenne muscular dystrophy (DMD) is characterized by se

161 Duchenne muscular dystrophy (DMD) is the most common and

162 Duchenne muscular dystrophy (DMD) is the most common inh

163 Patients with Duchenne muscular dystrophy (DMD) lack the protein dystr

164 Serum biomarkers in Duchenne muscular dystrophy (DMD) may provide deeper ins

165 h glucose enhances exon-skipping activity in Duchenne muscular dystrophy (DMD) mdx mice.

166 only show preclinical efficacy for heart in Duchenne muscular dystrophy (DMD) models but also improv

167 actone improves skeletal muscle pathology in Duchenne muscular dystrophy (DMD) mouse models.

168 ective of whether they presented with severe Duchenne muscular dystrophy (DMD) or milder Becker muscu

169 and is abnormally elevated in the muscle of Duchenne muscular dystrophy (DMD) patients and animal mo

170 myomiRs) are highly enriched in the serum of Duchenne Muscular Dystrophy (DMD) patients and dystrophi

171 In Duchenne muscular dystrophy (DMD) patients and the mouse

172 Duchenne muscular dystrophy (DMD) patients lack dystroph

173 ment with corticosteroids is recommended for Duchenne muscular dystrophy (DMD) patients to slow the p

174 presents a standard palliative treatment for Duchenne muscular dystrophy (DMD) patients, but various

175 -3 (MYOM3) are abnormally present in sera of Duchenne muscular dystrophy (DMD) patients, limb-girdle

176 biopsies from most independent FSHD, DM2 or Duchenne muscular dystrophy (DMD) studies compared to co

177 previously tested the implication of ApN in Duchenne muscular dystrophy (DMD) using mdx mice, a mode

178 is expected that serum protein biomarkers in Duchenne muscular dystrophy (DMD) will reflect disease p

179 o oligomer, enabled dystrophin production in Duchenne muscular dystrophy (DMD) with genetic mutations

180 tal muscle during the disease progression of Duchenne muscular dystrophy (DMD), a degenerative muscle

181 mon and severe form of muscular dystrophy is Duchenne muscular dystrophy (DMD), a disorder caused by

182 e athletes and poorly understood efficacy in Duchenne muscular dystrophy (DMD), a genetic muscle-wast

183 here is currently no effective treatment for Duchenne muscular dystrophy (DMD), a lethal monogenic di

184 rate these properties in two mouse models of Duchenne muscular dystrophy (DMD), a neurogenetic diseas

185 rophin gene to create mutations that lead to Duchenne muscular dystrophy (DMD), a recessive X-linked

186 Duchenne muscular dystrophy (DMD), caused by mutations a

187 ping is a promising therapeutic strategy for Duchenne muscular dystrophy (DMD), employing morpholino

188 independent from the pathogenic mutation: in Duchenne muscular dystrophy (DMD), for instance, age at

189 the emergence of experimental therapies for Duchenne muscular dystrophy (DMD), it is fundamental to

190 y has emerged as a leading cause of death in Duchenne muscular dystrophy (DMD), limited studies and t

191 In Duchenne muscular dystrophy (DMD), loss of dystrophin le

192 In Duchenne muscular dystrophy (DMD), loss of the membrane

193 g in healthy muscle, but in diseases such as Duchenne muscular dystrophy (DMD), microtubule alteratio

194 In the mdx mouse, a model of Duchenne muscular dystrophy (DMD), microtubules are most

195 Tongue weakness, like all weakness in Duchenne muscular dystrophy (DMD), occurs as a result of

196 Congenital neuromuscular disorders, such as Duchenne muscular dystrophy (DMD), spinal muscular atrop

197 of the muscle histopathology associated with Duchenne Muscular Dystrophy (DMD), the molecular and cel

198 In Duchenne muscular dystrophy (DMD), the reading frame of

199 ntial for treating genetic diseases, such as Duchenne muscular dystrophy (DMD), which is caused by mu

200 tion is a promising therapeutic strategy for Duchenne muscular dystrophy (DMD), which should be appli

201 phase 2 clinical study for the treatment of Duchenne muscular dystrophy (DMD).

202 are major contributors to muscle wasting in Duchenne muscular dystrophy (DMD).

203 to therapy are needed for clinical trials in Duchenne muscular dystrophy (DMD).

204 AAV) vectors hold great promise for treating Duchenne muscular dystrophy (DMD).

205 integrity and drive muscle deterioration in Duchenne muscular dystrophy (DMD).

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

207 unt of utrophin, a known disease modifier in Duchenne muscular dystrophy (DMD).

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

209 dystrophin are a possible means of treating Duchenne Muscular Dystrophy (DMD).

210 e, prevent dystrophin translation, and cause Duchenne muscular dystrophy (DMD).

211 biogenesis contributes to the progression of Duchenne muscular dystrophy (DMD).

212 of dystrophin-deficient mdx mice, a model of Duchenne muscular dystrophy (DMD).

213 lls carrying dystrophin mutations that cause Duchenne muscular dystrophy (DMD).

214 n great promise for exon-skipping therapy of Duchenne Muscular Dystrophy (DMD).

215 ardiomyopathy and mortality in patients with Duchenne muscular dystrophy (DMD).

216 ted to be an important pathogenic feature in Duchenne muscular dystrophy (DMD).

217 sidered a promising therapeutic approach for Duchenne muscular dystrophy (DMD).

218 in both preclinical models and subjects with Duchenne muscular dystrophy (DMD).

219 of cardiac disease in the mdx mouse model of Duchenne muscular dystrophy (DMD); however, a mechanisti

220 iously described in various animal models of Duchenne muscular dystrophy (DMD); however, the patholog

221 ed from fibroblasts of a patient affected by Duchenne Muscular Dystrophy (DMD, complete loss of dystr

222 muscles of mdx mice (i.e., a mouse model of Duchenne Muscular Dystrophy [DMD]) could restore the mor

223 In preclinical models for Duchenne muscular dystrophy, dystrophin restoration duri

224 ouse survival, and in the mdx mouse model of Duchenne muscular dystrophy, exosomes secreted by the en

225 the most common forms of muscular dystrophy: Duchenne muscular dystrophy, facioscapulohumeral muscula

226 pecific genetic conditions; Cystic Fibrosis, Duchenne Muscular Dystrophy, Familial Adenomatous Polypo

227 ation is sustained in the mdx mouse model of Duchenne muscular dystrophy for 1 year after a single in

228 Bisphosphonates have been used to treat Duchenne muscular dystrophy for prevention of osteoporos

229 of age; time to rise from floor </=7 s) with Duchenne muscular dystrophy from 13 specialist centres i

230 a 1-Mb contiguous region of the X-chromosome Duchenne muscular dystrophy gene.

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

232 luated for the treatment of diseases such as Duchenne muscular dystrophy, hemophilia, heart failure,

233 ich have a point mutation in Dmd)-a model of Duchenne muscular dystrophy-Hippo deficiency protected a

234 ment is recommended as a standard of care in Duchenne muscular dystrophy; however, few studies have a

235 Duchenne muscular dystrophy in boys progresses rapidly t

236 A cooperative effort of stakeholders in Duchenne muscular dystrophy-including representatives fr

237 In muscle lacking dystrophin, such as Duchenne muscular dystrophy, increased load during contr

238 In the degenerative disease Duchenne muscular dystrophy, inflammatory cells enter mu

239 Duchenne muscular dystrophy is a deadly muscle-wasting d

240 Duchenne muscular dystrophy is a fatal X-linked disease

241 Duchenne muscular dystrophy is a genetic disorder that s

242 Duchenne muscular dystrophy is a lethal genetic defect t

243 Duchenne muscular dystrophy is a lethal genetic disease

244 Duchenne muscular dystrophy is a progressive and incurab

245 Duchenne muscular dystrophy is a rare, progressive, musc

246 Duchenne muscular dystrophy is a severe and progressive

247 RATIONALE: Duchenne muscular dystrophy is a severe inherited form o

248 Duchenne muscular dystrophy is caused by dystrophin defi

249 king the protein dystrophin, as occurring in Duchenne muscular dystrophy, is a hypersensitivity to co

250 nts with Becker muscular dystrophy (BMD) and Duchenne muscular dystrophy lack neuronal nitric oxide s

251 the dystrophin-deficient mdx mouse model of Duchenne muscular dystrophy, limb muscles are especially

252 the development of cell-based therapies for Duchenne muscular dystrophy, little is known regarding t

253 cquired from blood serum of a mouse model of Duchenne muscular dystrophy (mdx) and control mice.

254 ct cardiac myocytes from the murine model of Duchenne muscular dystrophy (mdx) despite robust increas

255 mage in different muscular dystrophies (MDs; Duchenne muscular dystrophy, megaconial congenital muscu

256 ound that the haploinsufficiency of Dock3 in Duchenne muscular dystrophy mice improved dystrophic mus

257 blunting progressive chamber hypertrophy in Duchenne muscular dystrophy mice.

258 is in longitudinal studies in a large-animal Duchenne muscular dystrophy model in pigs, and then appl

259 -treated animal as well as in the C. elegans Duchenne muscular dystrophy model.

260 cued the muscle atrophy and dysfunction in a Duchenne muscular dystrophy mouse model.

261 for skeletal muscles in addition to heart in Duchenne muscular dystrophy mouse models and that minera

262 ophic phenotype in the mdx (a mouse model of Duchenne muscular dystrophy) mouse by blunting the regen

263 entres in the USA aged 7 years or older with Duchenne muscular dystrophy, myocardial damage by late g

264 In Duchenne muscular dystrophy myocytes (mdx/utrophin defic

265 uction of dystrophin expression in muscle of Duchenne muscular dystrophy patients by systemic adminis

266 Our data align the D2-mdx with Duchenne muscular dystrophy patients with the LTBP4 gene

267 ardiomyopathy is a leading cause of death in Duchenne muscular dystrophy patients, and currently no e

268 In mdx mice and Duchenne muscular dystrophy patients, dystrophin is lack

269 ha7beta1 integrin observed in mdx muscle and Duchenne muscular dystrophy patients.

270 onsistently, CD82 expression is decreased in Duchenne muscular dystrophy patients.

271 t in regenerating myofibers of patients with Duchenne muscular dystrophy, polymyositis, and compartme

272 lly deleting S1PR3 in the mdx mouse model of Duchenne muscular dystrophy produced a less severe muscl

273 In Duchenne muscular dystrophy, progressive loss of muscle

274 We were able to phase the Duchenne muscular dystrophy region into two contiguous h

275 therapy has shown great clinical promise in Duchenne muscular dystrophy, resulting in the production

276 s identified 6 unique oxidized proteins from Duchenne muscular dystrophy samples (n = 6) (versus cont

277 A limited number of DM2 and Duchenne muscular dystrophy samples were also sequenced.

278 The Duchenne muscular dystrophy sartorius muscle and ortholo

279 editing, CRISPR-Cas9, neuromuscular disease, Duchenne muscular dystrophy, spinal muscular atrophy, am

280 describing neuromuscular diseases, including Duchenne muscular dystrophy, spinal muscular atrophy, am

281 rs, including neuromuscular diseases such as Duchenne muscular dystrophy, spinal muscular atrophy, am

282 id steroids affects muscle remodeling in non-Duchenne muscular dystrophies, suggesting a positive out

283 For Duchenne muscular dystrophy, the rationale for exon skip

284 In Duchenne muscular dystrophy, this linkage system is abse

285 ively affected development of treatments for Duchenne muscular dystrophy; this approach could serve a

286 cal trials to treat genetic diseases such as Duchenne muscular dystrophy, we propose that exon skippi

287 the USA, patients (age 10-18 years old) with Duchenne muscular dystrophy were randomly assigned in a

288 cribed for chronic muscle conditions such as Duchenne muscular dystrophy, where their use is associat

289 Mutations in dystrophin lead to Duchenne muscular dystrophy, which is among the most com

290 For example, Duchenne muscular dystrophy, which is caused by mutation

291 spective case control study of patients with Duchenne muscular dystrophy who underwent serial cardiac

292 d safety of idebenone in young patients with Duchenne muscular dystrophy who were not taking concomit

293 K of 360 boys aged 3-15 years with confirmed Duchenne muscular dystrophy who were treated with daily

294 Patients with Duchenne muscular dystrophy with an LV ejection fraction

295 ively studied in mdx mice, a murine model of Duchenne muscular dystrophy with dystrophin deficiency.

296 n top of background therapy in patients with Duchenne muscular dystrophy with early myocardial diseas

297 els of Charcot-Marie-Tooth disease (CMT) and Duchenne muscular dystrophy, with no evidence of systemi

298 defective autophagy in mdx mice, a model of Duchenne muscular dystrophy, with the use of rapamycin-l

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

300 ing and universal small molecule therapy for Duchenne muscular dystrophy would be an enormous advance