ライフサイエンスコーパス: muscular atrophy

1 active survival motor neuron 2 (SMN2, spinal muscular atrophy).

2 y trial of nusinersen in infants with spinal muscular atrophy.

3 in that is deficient in patients with spinal muscular atrophy.

4 tal muscle regeneration for the treatment of muscular atrophy.

5 tisense drug being developed to treat spinal muscular atrophy.

6 nts) in patients with infantile-onset spinal muscular atrophy.

7 lls (iMNs) from a patient affected by spinal muscular atrophy.

8 tudy of nusinersen in infantile-onset spinal muscular atrophy.

9 icacy of nusinersen in adults with 5q spinal muscular atrophy.

10 as amyotrophic lateral sclerosis and spinal muscular atrophy.

11 cluding those involved in cancers and spinal muscular atrophy.

12 DcpS enzyme, a therapeutic target for spinal muscular atrophy.

13 as amyotrophic lateral sclerosis and spinal muscular atrophy.

14 euron protein (SMN) cause the disease spinal muscular atrophy.

15 sensory neuropathy type 1, and non-5q spinal muscular atrophy.

16 is, hereditary spastic paraplegia and spinal muscular atrophy.

17 a, amyotrophic lateral sclerosis, and spinal muscular atrophy.

18 ved in the pathogenesis of spinal and bulbar muscular atrophy.

19 lerosis was made; 4 patients had progressive muscular atrophy.

20 f arimoclomol in mice with spinal and bulbar muscular atrophy.

21 ssociated with motor neuron degeneration and muscular atrophy.

22 deficiency in the etiopathogenesis of spinal muscular atrophy.

23 e, amyotrophic lateral sclerosis, and spinal muscular atrophy.

24 hese were similar to controls in progressive muscular atrophy.

25 ing amyotrophic lateral sclerosis and spinal muscular atrophy.

26 ing postnatal nervous system, such as spinal muscular atrophy.

27 (survival of motor neuron protein) in spinal muscular atrophy.

28 of the neuromuscular disorder spinal bulbar muscular atrophy.

29 by recent successful interventions of spinal muscular atrophy.

30 n lower extremities or pelvic bones, 73% had muscular atrophy.

31 as been approved for the treatment of spinal muscular atrophy.

32 ot-Marie-Tooth disease and spinal and bulbar muscular atrophy.

33 may be one strategy in treating human spinal muscular atrophy.

34 bule motility in neurons may underlie spinal muscular atrophy.

35 is, spinal muscular atrophy, and spinobulbar muscular atrophy.

36 val motor neuron (SMN) protein causes spinal muscular atrophy, a neurodegenerative disease characteri

37 Reduced expression of SMN causes spinal muscular atrophy, a severe neurodegenerative disease.

38 atients with genetically confirmed 5q spinal muscular atrophy (age 16-65 years) with a homozygous del

39 sickle cell disease, cystic fibrosis, spinal muscular atrophy, alpha-thalassemia, and beta-thalassemi

40 ract associated with the disease spinobulbar muscular atrophy, also known as Kennedy disease.

41 Spinal and bulbar muscular atrophy, also known as Kennedy's disease, is an

42 disease, Duchenne muscular dystrophy, spinal muscular atrophy, amyotrophic lateral sclerosis, and myo

43 ncluding Duchenne muscular dystrophy, spinal muscular atrophy, amyotrophic lateral sclerosis, and myo

44 such as Duchenne muscular dystrophy, spinal muscular atrophy, amyotrophic lateral sclerosis, and myo

45 -Tooth type 2 (CMT2), scapuloperoneal spinal muscular atrophy and distal hereditary motor neuropathy

46 and dystrophin, genes responsible for spinal muscular atrophy and Duchenne muscular dystrophy, respec

47 e understanding of recessive proximal spinal muscular atrophy and how this is leading to exciting pot

48 ential in the treatment of spinal and bulbar muscular atrophy and may also be a possible approach for

49 e a therapeutic target for spinal and bulbar muscular atrophy and related polyglutamine diseases.

50 ogical disorder characterized by progressive muscular atrophy and respiratory failure.

51 ase characterized by motor neuron cell loss, muscular atrophy, and a shortened life span.

52 se mechanisms for myotonic dystrophy, spinal muscular atrophy, and fragile X syndrome, with broader i

53 ic dystrophy, mitochondrial myopathy, spinal muscular atrophy, and hereditary neuropathies.

54 treatment of inherited blindness and spinal muscular atrophy, and long-term therapeutic effects have

55 tions than high heel wear such as paralysis, muscular atrophy, and muscular dystrophy.

56 uch as amyotrophic lateral sclerosis, spinal muscular atrophy, and spinobulbar muscular atrophy.

57 arcot-Marie-Tooth disease type 2Z and spinal muscular atrophy, and the onset of symptoms ranges from

58 In children, inherited spinal muscular atrophies are the predominant diseases that aff

59 d in patients with a dominant form of spinal muscular atrophy, but how these mutations cause disease

60 ibroblasts derived from patients with spinal muscular atrophy by using bifunctional targeted oligonuc

61 h children and adults with a focus on spinal muscular atrophy, Charcot-Marie-Tooth disease and spinal

62 o adult care; (3) muscular dystrophy, spinal muscular atrophy, cystic fibrosis, haemophilia and sickl

63 tary causes are recognised, including spinal muscular atrophy, distal hereditary motor neuropathy and

64 ogressing and fatal disease characterized by muscular atrophy due to loss of upper and lower motor ne

65 95% CI, 0.08-0.10; P = .049) and progressive muscular atrophy (HR, 0.17; 95% CI, 0.22-1.36; P = .10).

66 receptor, causing X-linked spinal and bulbar muscular atrophy, impairs its function as a transcriptio

67 erference selectively in muscle cells caused muscular atrophy in larval stages and pupal lethality.

68 issue oedema, presence of synovial effusion, muscular atrophy in the affected extremity, osteopaenia,

69 Spinal muscular atrophy is a common motor neuron disease caused

70 Spinal muscular atrophy is a disorder of lower motor neurons, m

71 Spinal muscular atrophy is a fatal genetic disease of motoneuro

72 5q-Associated spinal muscular atrophy is a hereditary neuromuscular disease l

73 ication of virus-mediated GT to treat spinal muscular atrophy is a significant milestone, serving to

74 damage is detected in muscle cell nuclei and muscular atrophy is accelerated when one copy of the gen

75 Spinal muscular atrophy is an autosomal recessive neuromuscular

76 Spinal muscular atrophy is an inherited motor neuron disease th

77 Spinal muscular atrophy is an untreatable potentially fatal her

78 Spinal and bulbar muscular atrophy is an X-linked degenerative motor neuro

79 Spinal muscular atrophy is caused by loss of the survival motor

80 Spinal muscular atrophy is caused by reduced levels of SMN resu

81 ASOs to alter gene-splicing to treat spinal muscular atrophy is in phase 3 clinical trials.

82 Spinal muscular atrophy is the most common genetic killer of in

83 or (AR) causes Kennedy's disease/spinobulbar muscular atrophy (KD/SBMA) through poorly defined cellul

84 le in a number of diseases, including spinal muscular atrophy, leukemia, lymphoma, and breast cancer.

85 novo variants in BICD2 cause SMALED2 (spinal muscular atrophy lower extremity dominant 2), and a subs

86 Overall, the features of BICD2 spinal muscular atrophy, lower extremity predominant are consis

87 BICD2 spinal muscular atrophy, lower extremity predominant most commo

88 Spinal muscular atrophy, lower extremity-predominant, is charac

89 nd 7 women), and 4 patients with progressive muscular atrophy (mean age +/- SD, 59.2 +/- 5 y; 4 men).

90 Spinal and bulbar muscular atrophy mice that carry 100 pathogenic polyglut

91 bryonic motor neurons from spinal and bulbar muscular atrophy mice, which was accompanied by increase

92 e major gene products dysregulated in spinal muscular atrophy models in mice.

93 els of neuromuscular disease, such as spinal muscular atrophy, NMJ disorder and muscular dystrophy.

94 n is approved for the treatment of 5q spinal muscular atrophy of all types and stages in patients of

95 , for Duchenne muscular dystrophy and spinal muscular atrophy, offers hope not only for additional ne

96 ous familial hypercholesterolemia and spinal muscular atrophy) or as research tools to alter gene exp

97 role of AR proteolysis in spinal and bulbar muscular atrophy pathogenesis.

98 h nusinersen in a cohort of 85 type I spinal muscular atrophy patients of ages ranging from 2 months

99 In addition to the benefit to spinal muscular atrophy patients, there are discoveries from nu

100 esolving respiratory insufficiency at birth, muscular atrophy predominantly of the distal lower limbs

101 mutant mdf, which is affected by neurogenic muscular atrophy, progressive gait ataxia with tremor, c

102 the polyglutamine disease spinal and bulbar muscular atrophy, proteolysis of the mutant androgen rec

103 Spinal and bulbar muscular atrophy (SBMA) impairs motor function in men an

104 Spinal and bulbar muscular atrophy (SBMA) is a hereditary neuromuscular di

105 Spinal and bulbar muscular atrophy (SBMA) is a neuromuscular disease cause

106 Spinal and bulbar muscular atrophy (SBMA) is a neuromuscular disease chara

107 Spinal and bulbar muscular atrophy (SBMA) is a progressive neuromuscular d

108 Spinal bulbar muscular atrophy (SBMA) is a progressive, late onset neu

109 Spinobulbar muscular atrophy (SBMA) is an X-linked disease character

110 Spinal and bulbar muscular atrophy (SBMA) is an X-linked motoneuron diseas

111 Spinal and bulbar muscular atrophy (SBMA) is caused by polyglutamine expan

112 X-linked spinal and bulbar muscular atrophy (SBMA) is characterized by adult-onset

113 Spinal and bulbar muscular atrophy (SBMA) is characterized by loss of moto

114 Spinal and bulbar muscular atrophy (SBMA) results from a CAG repeat expans

115 receptor (AR) is responsible for spinobulbar muscular atrophy (SBMA) that leads to selective loss of

116 Here we consider X-linked spinal and bulbar muscular atrophy (SBMA), a repeat disorder caused by pol

117 Spinal and bulbar muscular atrophy (SBMA), an adult-onset neurodegenerativ

118 rogen receptor (AR) causes spinal and bulbar muscular atrophy (SBMA), an X-linked neuromuscular disea

119 ar atrophy (SMA), X-linked spinal and bulbar muscular atrophy (SBMA), and amyotrophic lateral scleros

120 a protein associated with spinal and bulbar muscular atrophy (SBMA), and the nuclear protein PTIP (P

121 degeneration in individuals with spinobulbar muscular atrophy (SBMA).

122 anded AR proteotoxicity in spinal and bulbar muscular atrophy (SBMA).

123 Spinal and bulbar muscular atrophy (SBMA, also known as Kennedy's disease)

124 s in the lethal motor neuron diseases spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis

125 Spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis

126 SC3 cause pontocerebellar hypoplasia, spinal muscular atrophy (SMA) and central nervous system demyel

127 ative conditions of childhood such as spinal muscular atrophy (SMA) and neuronal ceroid lipofuscinosi

128 A number of mouse models for spinal muscular atrophy (SMA) have been genetically engineered

129 dentified as a protective modifier of spinal muscular atrophy (SMA) in some patient populations and a

130 Spinal muscular atrophy (SMA) is a common and often fatal neuro

131 Spinal muscular atrophy (SMA) is a common neuromuscular disorde

132 The underlying cause of spinal muscular atrophy (SMA) is a deficiency of the survival m

133 Spinal muscular atrophy (SMA) is a devastating infantile geneti

134 Spinal muscular atrophy (SMA) is a devastating neuromuscular di

135 Feline spinal muscular atrophy (SMA) is a fully penetrant, autosomal r

136 Spinal muscular atrophy (SMA) is a genetic disease caused by mu

137 Spinal muscular atrophy (SMA) is a genetic disease characterize

138 Spinal muscular atrophy (SMA) is a genetic disorder caused by a

139 Spinal muscular atrophy (SMA) is a genetic disorder characteriz

140 Spinal muscular atrophy (SMA) is a hereditary neurodegenerative

141 Spinal muscular atrophy (SMA) is a lethal human disease charact

142 Spinal muscular atrophy (SMA) is a major inherited cause of inf

143 Spinal Muscular Atrophy (SMA) is a monogenic neurodegenerative

144 Spinal muscular atrophy (SMA) is a motoneuron disease caused by

145 Spinal muscular atrophy (SMA) is a motor neuron disease caused

146 Spinal muscular atrophy (SMA) is a motor neuron disease caused

147 Spinal muscular atrophy (SMA) is a motor neuron disease.

148 Spinal muscular atrophy (SMA) is a neurodegenerative disease ca

149 Spinal muscular atrophy (SMA) is a neurodegenerative disease ca

150 Spinal muscular atrophy (SMA) is a neurodegenerative disease ca

151 Spinal muscular atrophy (SMA) is a neurodegenerative disease ca

152 Spinal muscular atrophy (SMA) is a neurodegenerative disease ca

153 Spinal muscular atrophy (SMA) is a neurodegenerative disease ch

154 Spinal muscular atrophy (SMA) is a neurodegenerative disease pr

155 otor phenotype.SIGNIFICANCE STATEMENT Spinal muscular atrophy (SMA) is a neurodegenerative disease, c

156 Spinal muscular atrophy (SMA) is a neuromuscular disease caused

157 Spinal muscular atrophy (SMA) is a neuromuscular disease caused

158 Spinal muscular atrophy (SMA) is a neuromuscular disease caused

159 Spinal muscular atrophy (SMA) is a neuromuscular disease caused

160 Spinal muscular atrophy (SMA) is a neuromuscular disease caused

161 Spinal muscular atrophy (SMA) is a neuromuscular disease causin

162 Spinal muscular atrophy (SMA) is a neuromuscular disease charac

163 Spinal muscular atrophy (SMA) is a neuromuscular disease charac

164 Spinal muscular atrophy (SMA) is a neuromuscular disorder cause

165 Spinal Muscular Atrophy (SMA) is a neuromuscular disorder cause

166 Spinal muscular atrophy (SMA) is a neuromuscular disorder cause

167 Spinal muscular atrophy (SMA) is a pediatric neuromuscular cond

168 Spinal muscular atrophy (SMA) is a progressive motor neuron dis

169 Spinal muscular atrophy (SMA) is a progressive neurodegenerativ

170 Spinal muscular atrophy (SMA) is a progressive neurodegenerativ

171 Spinal Muscular Atrophy (SMA) is an autosomal recessive disorde

172 Spinal muscular atrophy (SMA) is an autosomal recessive disorde

173 Spinal muscular atrophy (SMA) is an autosomal recessive motor n

174 Spinal muscular atrophy (SMA) is an autosomal recessive neurode

175 Spinal muscular atrophy (SMA) is an autosomal recessive neurode

176 Spinal muscular atrophy (SMA) is an autosomal recessive neuromu

177 Spinal muscular atrophy (SMA) is an autosomal-recessive disorde

178 Spinal muscular atrophy (SMA) is an autosomal-recessive disorde

179 Spinal muscular atrophy (SMA) is an autosomal-recessive motor n

180 Spinal muscular atrophy (SMA) is an autosomal-recessive pediatr

181 Spinal muscular atrophy (SMA) is an inherited motor neuron dise

182 tor neuron (MN) degenerative disease, spinal muscular atrophy (SMA) is caused by deficiency of SMN (s

183 Spinal muscular atrophy (SMA) is caused by deficiency of SMN pr

184 The inherited motor neuron disease spinal muscular atrophy (SMA) is caused by deficient expression

185 BACKGROUNDSpinal muscular atrophy (SMA) is caused by deficient expression

186 Spinal muscular atrophy (SMA) is caused by deletions or mutatio

187 Spinal muscular atrophy (SMA) is caused by deletions or mutatio

188 Spinal muscular atrophy (SMA) is caused by depletion of the ubi

189 Spinal Muscular Atrophy (SMA) is caused by diminished Survival

190 Spinal muscular atrophy (SMA) is caused by homozygous mutations

191 Spinal muscular atrophy (SMA) is caused by loss of the survival

192 Spinal muscular atrophy (SMA) is caused by loss-of-function mut

193 Spinal muscular atrophy (SMA) is caused by low levels of surviv

194 The motoneuron disease spinal muscular atrophy (SMA) is caused by low levels of the su

195 Spinal muscular atrophy (SMA) is caused by mutation of the Surv

196 Spinal muscular atrophy (SMA) is caused by mutation or deletion

197 Spinal muscular atrophy (SMA) is caused by mutation or deletion

198 Spinal Muscular Atrophy (SMA) is caused by mutation or deletion

199 Spinal muscular atrophy (SMA) is caused by mutations in the SMN

200 Spinal muscular atrophy (SMA) is caused by reduced levels of su

201 The motor neuron disease spinal muscular atrophy (SMA) is caused by reduced levels of th

202 pproach to SMA.SIGNIFICANCE STATEMENT Spinal muscular atrophy (SMA) is caused by the loss of motor ne

203 Spinal muscular atrophy (SMA) is caused by the loss or mutation

204 Spinal muscular atrophy (SMA) is caused by the low levels of su

205 While spinal muscular atrophy (SMA) is characterized by motor neuron

206 Spinal muscular atrophy (SMA) is characterized by the selective

207 ity of the neurodegenerative disorder spinal muscular atrophy (SMA) is dependent on the levels of fun

208 Spinal Muscular Atrophy (SMA) is due to the loss of the surviva

209 Spinal muscular atrophy (SMA) is one of the most common severe

210 A pathological hallmark of spinal muscular atrophy (SMA) is severe motor neuron (MN) loss,

211 Spinal muscular atrophy (SMA) is the leading genetic cause of i

212 Spinal muscular atrophy (SMA) is the leading genetic cause of i

213 Infantile-onset spinal muscular atrophy (SMA) is the most common genetic cause

214 Proximal spinal muscular atrophy (SMA) is the most frequent cause of her

215 Spinal muscular atrophy (SMA) is the number 1 genetic killer of

216 d neuromuscular junction pathology of spinal muscular atrophy (SMA) mice.

217 and structure of spinal circuitry in spinal muscular atrophy (SMA) model mice.

218 sus Statement for Standard of Care in Spinal Muscular Atrophy (SMA) notes that patients suffer from g

219 Spinal muscular atrophy (SMA) occurs as a result of cell-ubiqui

220 activation of SMN2 exon 7 splicing in spinal muscular atrophy (SMA) patient fibroblasts, suggesting a

221 myotonic dystrophy type 1 (CDM1) and spinal muscular atrophy (SMA) patients.

222 Spinal muscular atrophy (SMA) presents severe muscle weakness w

223 Clinical presentation of spinal muscular atrophy (SMA) ranges from a neonatal-onset, ver

224 Spinal muscular atrophy (SMA) remains one of the most common an

225 l recessive neurodegenerative disease spinal muscular atrophy (SMA) results from low levels of surviv

226 asuring SMN1 and SMN2 copy numbers in spinal muscular atrophy (SMA) samples has not been reported.

227 Spinal muscular atrophy (SMA) type 0 is the most severe form of

228 ed in iPSC-derived motor neurons from Spinal Muscular Atrophy (SMA) type 1 patient's.

229 n a large cohort of 199 patients with spinal muscular atrophy (SMA) type III assessed using the Hamme

230 ron 1 (SMN1) protein in humans causes Spinal Muscular Atrophy (SMA), a debilitating childhood disease

231 in the SMN1 gene are associated with spinal muscular atrophy (SMA), a devastating neurodegenerative

232 Spinal muscular atrophy (SMA), a devastating neurodegenerative

233 Spinal muscular atrophy (SMA), a heritable neurodegenerative di

234 Spinal muscular atrophy (SMA), a leading genetic disease of chi

235 xon 7, skipping of which is linked to spinal muscular atrophy (SMA), a leading genetic disease of chi

236 Spinal muscular atrophy (SMA), a motoneuron disease caused by a

237 uced expression of SMN protein causes spinal muscular atrophy (SMA), a neurodegenerative disorder lea

238 Spinal muscular atrophy (SMA), a recessive neurodegenerative di

239 Spinal muscular atrophy (SMA), a recessive neuromuscular disord

240 ere we show that, in a mouse model of spinal muscular atrophy (SMA), a reduction in proprioceptive sy

241 evidence of IGF-1 axis alteration in spinal muscular atrophy (SMA), a very severe neurodegenerative

242 val Motor Neuron 1 (SMN1) gene causes spinal muscular atrophy (SMA), an autosomal recessive neurodege

243 Spinal muscular atrophy (SMA), an autosomal recessive neuromusc

244 Spinal muscular atrophy (SMA), an inherited disease of motor ne

245 een amyotrophic lateral sclerosis and spinal muscular atrophy (SMA), and 3 mutations of the ASC-1 gen

246 as Duchenne muscular dystrophy (DMD), spinal muscular atrophy (SMA), and Pompe disease (acid maltase

247 ng as a prominent systemic feature of spinal muscular atrophy (SMA), but little is known about the un

248 motor neuron (SMN) deficiency causes spinal muscular atrophy (SMA), but the pathogenesis mechanisms

249 nt/childhood onset motoneuron disease spinal muscular atrophy (SMA), caused by low levels of the surv

250 fish models of the motoneuron disease spinal muscular atrophy (SMA), motor axons fail to form the nor

251 Spinal muscular atrophy (SMA), the leading genetic cause of inf

252 Spinal muscular atrophy (SMA), the leading genetic cause of inf

253 Spinal muscular atrophy (SMA), the leading genetic disorder of

254 Homozygous loss of SMN1 causes spinal muscular atrophy (SMA), the most common and devastating

255 The neuromuscular disorder spinal muscular atrophy (SMA), the most common inherited killer

256 Homozygous SMN1 loss causes spinal muscular atrophy (SMA), the most common lethal genetic c

257 SMN) gene underlie the development of spinal muscular atrophy (SMA), which currently represents the l

258 f cell-cell interaction at the NMJ in spinal muscular atrophy (SMA), X-linked spinal and bulbar muscu

259 hich causes the neuromuscular disease spinal muscular atrophy (SMA)-binds to ribosomes and that this

260 -Gemin2 interaction is abrogated by a spinal muscular atrophy (SMA)-causing mutation in an SMN helix

261 y for neuromuscular diseases, such as spinal muscular atrophy (SMA).

262 is found in patients with the disease spinal muscular atrophy (SMA).

263 l motor neuron-1 (SMN1) protein cause spinal muscular atrophy (SMA).

264 c mice are well-established models of spinal muscular atrophy (SMA).

265 of motor neuron (SMN) protein lead to spinal muscular atrophy (SMA).

266 ifier of the human motoneuron disease spinal muscular atrophy (SMA).

267 e result in the motor neuron disease, spinal muscular atrophy (SMA).

268 ting childhood neuromuscular disease, spinal muscular atrophy (SMA).

269 nfantile-onset motor neuron disorder, spinal muscular atrophy (SMA).

270 to neurodegenerative diseases such as spinal muscular atrophy (SMA).

271 oving towards a potential therapy for spinal muscular atrophy (SMA).

272 es motor function in a mouse model of spinal muscular atrophy (SMA).

273 en two motor neuron diseases, ALS and spinal muscular atrophy (SMA).

274 hallmark of the neuromuscular disease spinal muscular atrophy (SMA); however, it is unclear whether t

275 om patients with types I, II, and III spinal muscular atrophy (SMA-I, -II, and -III, respectively).

276 del of the devastating human disease "spinal muscular atrophy" (SMA) was used to investigate the seve

277 Spinal muscular atrophies (SMAs) are a heterogeneous group of i

278 weeks and 7 months old with onset of spinal muscular atrophy symptoms between 3 weeks and 6 months,

279 Secondary radiological findings, such as muscular atrophy, synovitis, posture-gait deterioration,

280 l subjects and all patients with progressive muscular atrophy tested, but not in the patients with pr

281 Using a mouse model of spinal and bulbar muscular atrophy that exhibits many of the characteristi

282 ival motor neuron (SMN) protein cause spinal muscular atrophy, the leading genetic disorder for infan

283 In progressive muscular atrophy, there is selective degeneration of ant

284 Among infants with spinal muscular atrophy, those who received nusinersen were mor

285 sclerosis and six patients with progressive muscular atrophy, together with 16 age-matched controls.

286 Spinal muscular atrophy type 1 (SMA1) is a progressive, monogen

287 n resting energy expenditure (REE) in spinal muscular atrophy type I (SMAI) is still limited.

288 MT2D from the allelic disorder distal spinal muscular atrophy type V.

289 A pronounced muscular atrophy was detected in the esophagus and colon

290 In 6% of them muscular atrophy was severe, and they had posture-gait d

291 treat Duchenne muscular dystrophy and spinal muscular atrophy, which are currently being tested in cl

292 ality control may also be relevant in spinal muscular atrophy, which is caused by defects in the surv

293 ions in cortical development (MCD) or spinal muscular atrophy with lower extremity predominance (SMAL

294 Loa) (DYNC1H1(F580Y)) mouse model for spinal muscular atrophy with lower extremity predominance and a

295 (DYNC1H1) of cytoplasmic dynein cause spinal muscular atrophy with lower extremity predominance, Char

296 distal hereditary motor neuropathies, spinal muscular atrophy with parkinsonism and the later stages

297 Spinal muscular atrophy with pontocerebellar hypoplasia (SMA-PC

298 IGHMBP2 mutations usually lead to spinal muscular atrophy with respiratory distress type 1 (SMARD

299 en in the treatment of adults with 5q spinal muscular atrophy, with clinically meaningful improvement

300 in amyotrophic lateral sclerosis and spinal muscular atrophy, yet its function in the nervous system