ライフサイエンスコーパス: enzyme replacement therapy

1 s after surgery without requiring pancreatic enzyme replacement therapy.

2 t LPL have thus far prevented development of enzyme replacement therapy.

3 ollow-up, medical management, and the use of enzyme replacement therapy.

4 al protein nitration that is reversible with enzyme replacement therapy.

5 istopathological improvements observed after enzyme replacement therapy.

6 n of activity in peripheral bone marrow with enzyme replacement therapy.

7 age accumulated gradually after cessation of enzyme replacement therapy.

8 active approach for cellular-based, systemic enzyme replacement therapy.

9 otein, suggesting they could be suitable for enzyme replacement therapy.

10 of contributing to the development of an LPL enzyme replacement therapy.

11 improve the therapeutic efficacy of MPS IVA enzyme replacement therapy.

12 ulation in tissues poorly treated by current enzyme replacement therapy.

13 inuric mice on methionine-restricted diet or enzyme replacement therapy.

14 They can be viewed as enzyme replacement therapy.

15 ear safety and efficacy of asfotase alfa, an enzyme replacement therapy.

16 exocrine pancreatic insufficiency requiring enzyme replacement therapy.

17 of N-linked glycoproteins and can be used in enzyme replacement therapy.

18 (rhASA) is currently under development as an enzyme replacement therapy.

19 rosis and for reduced or delayed response to enzyme replacement therapy.

20 ed by reducing heparan sulfate storage using enzyme replacement therapy.

21 ical intervention, and the use of pancreatic enzyme-replacement therapy.

22 st often treated postnatally with protein or enzyme replacement therapies.

23 study of digestive processes and pancreatic enzyme replacement therapies.

24 al biopsies at baseline and after 5 years of enzyme replacement therapy; 7 patients had additional bi

25 We have also noted that, in the absence of enzyme replacement therapy, absolute neutrophil counts o

26 ystemic disorder, treatable with recombinant enzyme replacement therapy (agalsidase).

27 Enzyme replacement therapies and pre-clinical studies on

28 ncern, podocytes are relatively resistant to enzyme replacement therapy and are poorly replicating, w

29 Although enzyme replacement therapy and bone marrow transplantati

30 Therapeutic modalities such as enzyme replacement therapy and gene therapy are not idea

31 here allow for predictable outcomes of ENPP1 enzyme replacement therapy and provide plausible expecta

32 B has potential for intracerebrospinal fluid enzyme replacement therapy and should be further explore

33 Enzyme replacement therapy and substrate reduction thera

34 and also provide useful models for studying enzyme replacement therapy and targeted correction of mi

35 nt at 6 months have continued not to receive enzyme-replacement therapy and have had stable gene mark

36 ieve protein expression as an alternative to enzyme replacement therapies, and to express chimeric an

37 ases, including bone marrow transplantation, enzyme replacement therapy, and gene therapy.

38 ency in vitro establishes the feasibility of enzyme replacement therapy, and has important implicatio

39 c stem-cell transplantation, reinitiation of enzyme-replacement therapy, and additional gene therapy)

40 tment options for Fabry disease, recombinant enzyme replacement therapy (approved in the United State

41 Impediments to enzyme replacement therapy are the absence of mannose 6-

42 re comparison with the standard treatment of enzyme replacement therapy as well as longer-term follow

43 All patients received enzyme replacement therapy at the time of evaluation.

44 During efforts to develop an enzyme replacement therapy based on a recombinant form o

45 ase alfa (Brineura), a tripeptidyl peptidase enzyme replacement therapy, became the first globally ap

46 patient outcomes as care standards including enzyme replacement therapy can be applied and complicati

47 Biologic drugs, including enzyme-replacement therapies, can elicit anti-drug Abs (

48 st that intermittent intracerebroventricular enzyme replacement therapy dosing with rhbeta-Gal is a t

49 A regimen of intermittent enzyme replacement therapy dosing with rhbeta-Gal, follo

50 nation with maintenance dose of imiglucerase enzyme replacement therapy during 1 year of treatment in

51 Such patients can receive enzyme replacement therapy during which a human placenta

52 Following the introduction of enzyme replacement therapy, early diagnosis and treatmen

53 ease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE), at 1 year, eliglust

54 ere are reports of AVN in patients receiving enzyme replacement therapy (ERT) , and it is not known w

55 m 34 untreated and 33 Fabry males treated by enzyme replacement therapy (ERT) and 54 untreated and 19

56 of gene-modified cells is an alternative to enzyme replacement therapy (ERT) and allogeneic HSCT tha

57 Enzyme replacement therapy (ERT) and hematopoietic stem

58 increase GCase activity in lysosomes involve enzyme replacement therapy (ERT) and molecular chaperone

59 Here, an enzyme replacement therapy (ERT) approach in fibroblasts

60 Here, an enzyme replacement therapy (ERT) approach in fibroblasts

61 We therefore tested the efficacy of enzyme replacement therapy (ERT) by administering monthl

62 (AAV8-LSPhGAA) could eliminate the need for enzyme replacement therapy (ERT) by creating a liver dep

63 Preclinical studies of enzyme replacement therapy (ERT) by using two GALNS enzy

64 ons (WMLs) and brain infarctions and whether enzyme replacement therapy (ERT) changes this risk.

65 Enzyme replacement therapy (ERT) effectively reverses st

66 ted in a highly-sensitized patient receiving enzyme replacement therapy (ERT) for Pompe disease, but

67 Enzyme replacement therapy (ERT) for the lysosomal stora

68 seful adjunctive therapy in combination with enzyme replacement therapy (ERT) for the treatment of GD

69 (ASMKO) of NPD, we evaluated the efficacy of enzyme replacement therapy (ERT) for the treatment of th

70 Enzyme replacement therapy (ERT) for type 1 Gaucher has

71 Enzyme replacement therapy (ERT) has been shown to be ef

72 Enzyme replacement therapy (ERT) has been shown to be ef

73 Cardiac response to enzyme replacement therapy (ERT) in Fabry disease is typ

74 Assessing the skeletal response to enzyme replacement therapy (ERT) in Gaucher disease (GD)

75 Effective dosages for enzyme replacement therapy (ERT) in Pompe disease are mu

76 rough biweekly intracerebroventricular (ICV) enzyme replacement therapy (ERT) involving recombinant h

77 Together, these data suggest that CBS enzyme replacement therapy (ERT) is a promising approach

78 Enzyme replacement therapy (ERT) is a standard therapeut

79 Enzyme replacement therapy (ERT) is a treatment option f

80 Enzyme replacement therapy (ERT) is available for severa

81 Although enzyme replacement therapy (ERT) is considered standard

82 While enzyme replacement therapy (ERT) is the only effective t

83 Enzyme replacement therapy (ERT) is the only treatment f

84 investigate the effectiveness of intravenous enzyme replacement therapy (ERT) on corneal GAG accumula

85 cumulation of globotriaosylceramide (Gb3) by enzyme replacement therapy (ERT) or chaperone-mediated s

86 Enzyme replacement therapy (ERT) partially and temporari

87 JCI, Bublil and colleagues demonstrate that enzyme replacement therapy (ERT) provides long-term amel

88 o-Gb(3) analogue levels correlated well with enzyme replacement therapy (ERT) status in males (p < 0.

89 ecrease significantly after the beginning of enzyme replacement therapy (ERT) treatment and remain st

90 Enzyme replacement therapy (ERT) was eventually shown to

91 The only approved treatment is enzyme replacement therapy (ERT) with human recombinant

92 PD is currently treated with enzyme replacement therapy (ERT) with intravenous infusi

93 2 other options are available for ADA-SCID: enzyme replacement therapy (ERT) with pegylated bovine A

94 Enzyme replacement therapy (ERT) with recombinant GAA is

95 The efficacy of enzyme replacement therapy (ERT) with recombinant human

96 cy (ADA SCID), which can be treated with ADA enzyme replacement therapy (ERT), allogeneic hematopoiet

97 with systemic recombinant human GAA (rhGAA) enzyme replacement therapy (ERT), but the current standa

98 Keywords: Fabry Disease, Enzyme Replacement Therapy (ERT), Cardiac MRI, Late Gado

99 All patients should initially receive enzyme replacement therapy (ERT), followed by definitive

100 Treatments include enzyme replacement therapy (ERT), hematopoietic cell tra

101 The current standard treatment, enzyme replacement therapy (ERT), is not curative and ha

102 Since the advent of enzyme replacement therapy (ERT), the clinical outcomes

103 Enzyme replacement therapy (ERT), where recombinant enzy

104 To a degree, Fabry disease is manageable via enzyme replacement therapy (ERT).

105 itute a functioning immune system, including enzyme replacement therapy (ERT).

106 e Fabry patients, which was not corrected by enzyme replacement therapy (ERT).

107 disease type 1 is alternate-week infusion of enzyme replacement therapy (ERT).

108 ersus receiving standard non-BBB penetrating enzyme replacement therapy (ERT, n = 12).

109 After many years of intensive investigation, enzyme-replacement therapy (ERT) has become standard tre

110 the safety and efficacy results of in utero enzyme-replacement therapy (ERT) in a fetus with CRIM (c

111 Although continued enzyme-replacement therapy (ERT) in combination with die

112 given nonmyeloablative conditioning and ADA enzyme-replacement therapy (ERT) is withheld before auto

113 plantation cytoreduction and remained on ADA enzyme-replacement therapy (ERT) throughout the procedur

114 ed the clinical and biochemical responses to enzyme-replacement therapy (ERT) with macrophage-targete

115 Current enzyme replacement therapies (ERTs) face limited effecti

116 Some current enzyme replacement therapies (ERTs) for lysosomal storag

117 Rats receiving either bacteria or enzyme replacement therapy excreted far lower levels of

118 The combination of enzyme replacement therapy followed by BMT reduced lysos

119 t identical molecules that were developed as enzyme replacement therapies for Gaucher disease.

120 Thus, enzyme replacement therapy for 30-36 mo with agalsidase

121 In recent studies of enzyme replacement therapy for animal models with lysoso

122 Since the development of enzyme replacement therapy for CLN2 disease, much has be

123 fucosidosis, and the mucopolysaccharidoses; enzyme replacement therapy for fucosidosis, the mucopoly

124 Enzyme replacement therapy for Gaucher disease is costly

125 Alglucerase, a macrophage-targeted enzyme replacement therapy for Gaucher disease, has been

126 With the advent of a new enzyme replacement therapy for GSD II, there is a need f

127 which is important since clinical trials of enzyme replacement therapy for LAL deficiency are curren

128 Enzyme replacement therapy for lysosomal storage disease

129 Enzyme replacement therapy for lysosomal storage disorde

130 tion and in patients who have been receiving enzyme replacement therapy for more than 2 years, as sug

131 These results suggest the feasibility of enzyme replacement therapy for MPS IIIB.

132 Enzyme replacement therapy for neuronopathic MPS require

133 Clinical studies of enzyme replacement therapy for Pompe disease have indica

134 rhGAA and therefore the clinical efficacy of enzyme replacement therapy for Pompe disease may be impr

135 LT-tagged GAA enzyme may provide an improved enzyme replacement therapy for Pompe disease patients.

136 take and delivery of enzymes to lysosomes in enzyme replacement therapy for the treatment of lysosoma

137 eat promise as a platform for cellular-based enzyme replacement therapy for the treatment of mucopoly

138 mbinant human tripeptidyl peptidase 1 (TPP1) enzyme replacement therapy for the treatment of neuronal

139 To investigate the mechanisms underlying enzyme replacement therapy for this disorder, we studied

140 be useful as non-immunogenic alternatives in enzyme replacement therapy for treatment of lysosomal st

141 ndent lysosomal targeting system may enhance enzyme-replacement therapy for certain human lysosomal s

142 Preclinical studies of enzyme-replacement therapy for Fabry disease (deficient

143 dy provides the basis for a phase 3 trial of enzyme-replacement therapy for Fabry disease.

144 These targeted therapies include enzyme replacement therapies, gene therapies targeting t

145 d therapies are being developed that include enzyme replacement therapy, gene therapy, and substrate

146 They demonstrate that ADA patients receiving enzyme replacement therapy had B cell tolerance checkpoi

147 Enzyme replacement therapy has been proposed, but furthe

148 Enzyme replacement therapy has been used successfully in

149 Enzyme replacement therapy has been used to treat mucopo

150 Enzyme replacement therapy has potential benefit but has

151 Enzyme replacement therapies have revolutionized patient

152 g the residual activity of a missing enzyme (enzyme replacement therapy, hematopoietic stem cell tran

153 nging from ex vivo cellular manipulations to enzyme replacement therapies in humans.

154 Enzyme replacement therapy in all males with Fabry disea

155 eduction therapy can improve the efficacy of enzyme replacement therapy in cell culture and in mice.

156 alysed 5-year treatment with agalsidase alfa enzyme replacement therapy in patients with Fabry's dise

157 hanges in reticuloendothelial activity after enzyme replacement therapy in patients with Gaucher dise

158 xpression of this BBB transporter would make enzyme replacement therapy in the adult possible.

159 With the development of enzyme replacement therapy in the past few years, early

160 In summary, long-term enzyme replacement therapy in young patients can result

161 We conducted a phase 3 trial of enzyme-replacement therapy in children and adults with l

162 om birth to the first clinical visit (before enzyme replacement therapy) in 499 adult patients (mean

163 Enzyme replacement therapy is being developed for the la

164 Enzyme replacement therapy is currently available for th

165 B) prevents enzymes from reaching the brain, enzyme replacement therapy is effective only against the

166 deficits can be prevented in MPS VII mice if enzyme replacement therapy is initiated early in life.

167 Therefore, enzyme replacement therapy is not feasible using current

168 Enzyme replacement therapy is one therapeutic option, bu

169 Enzyme replacement therapy is the principal treatment fo

170 Enzyme-replacement therapy is an established means of tr

171 Despite the benefits of enzyme replacement therapy, it has limitations-most impo

172 pact of augmentative recombinant intravenous enzyme replacement therapy (IV-ERT) post transplantation

173 th Fabry's disease who were not treated with enzyme replacement therapy, long-term treatment with aga

174 In conclusion, before treatment with enzyme replacement therapy, men with classical Fabry dis

175 lucosidase alfa for at least 2 years or were enzyme replacement therapy-naive.

176 Enzyme replacement therapy normalized the caloric densit

177 nse to the enzyme alpha-l-iduronidase during enzyme replacement therapy of a canine model of the lyso

178 Enzyme replacement therapy often falls short of adequate

179 ew and meta-analysis to assess the effect of enzyme replacement therapy on cardiac MRI parameters in

180 The effect of early-onset enzyme replacement therapy on renal morphologic features

181 ing the natural history period (i.e., before enzyme replacement therapy or among patients who never r

182 survival (in the absence of reinitiation of enzyme-replacement therapy or rescue allogeneic hematopo

183 n HCM mimic led to change in management (eg, enzyme replacement therapy) or family screening in all c

184 matopoietic stem cell transplantation (HCT), enzyme replacement therapy, or gene therapy for SCID and

185 cal hematopoietic stem cell transplantation, enzyme replacement therapy, or gene therapy.

186 lism and immune functions can be achieved by enzyme replacement therapy, or more effectively by bone

187 and discuss available treatments, including enzyme replacement therapy, oral lipid-lowering therapy,

188 findings have important implications for NPD enzyme replacement therapy, particularly in the lung.

189 ent patients with late-onset forms and after enzyme replacement therapy (PEG-ADA) are known to manife

190 Oral pancreatic enzyme replacement therapy (PERT) with pancreatin produc

191 therapy, palliative chemotherapy, pancreatic enzyme replacement therapy (PERT), referral to a dietici

192 of comorbidities, and the use of pancreatic enzyme replacement therapy (PERT).

193 tration required manufacturers of pancreatic enzymes replacement therapy (PERT) to have approval for

194 atment of individual symptoms in addition to enzyme replacement therapy seems to be needed for many p

195 e levels in GD1 patients, which decline upon enzyme-replacement therapy; serum ceramide levels remain

196 f GCase-based therapeutics, such as gene and enzyme replacement therapies, small molecule chaperones

197 valglucosidase alfa, a recombinant human GAA enzyme replacement therapy specifically designed for enh

198 ratified by 6-min walk distance and previous enzyme replacement therapy status, to intravenous cipagl

199 Treatments include enzyme replacement therapy, stem/progenitor cell transpl

200 Notably, enzyme replacement therapy (the only available therapy f

201 ons are available for some diseases, such as enzyme replacement therapy to correct enzyme deficiency

202 harnessed to create a cellular reservoir of enzyme replacement therapy to diseased brain.

203 mice were treated with various levels of ADA enzyme replacement therapy to regulate endogenous adenos

204 ant form of human ASM, is being developed as enzyme replacement therapy to treat the non-neurological

205 bers isolated from wild-type, untreated, and enzyme replacement therapy-treated GAA knock-out mice.

206 correlate these results with patient gender, enzyme replacement therapy treatment, and lyso-Gb3 analo

207 to evaluate the long-term impact of a novel enzyme replacement therapy [truncated human CBS C15S mut

208 ) might be for specific lysosomal disorders (enzyme replacement therapy via intrathecal or intracereb

209 ration rate versus baseline after 5 years of enzyme replacement therapy was -3.17 mL/min per 1.73 m(2

210 Conclusion In patients with Fabry disease, enzyme replacement therapy was associated with stabiliza

211 e been an area of interest since intravenous enzyme replacement therapy was successfully introduced f

212 ENB-0040, an enzyme-replacement therapy, was associated with improved

213 aceuticals such as monoclonal antibodies and enzyme replacement therapies, which are largely excluded

214 valuable data for the further development of enzyme replacement therapy, which is currently the only

215 Enzyme replacement therapy with a soluble ENPP1 biologic

216 Pompe disease has resisted enzyme replacement therapy with acid alpha-glucosidase (

217 nts who have Fabry disease and also received enzyme replacement therapy with agalsidase-beta, given a

218 ticotropic hormone for infantile spasms, and enzyme replacement therapy with alglucosidase alpha for

219 , and spleen provided evidence that in utero enzyme replacement therapy with GUS-Fc targeted sites of

220 It paved the way for the development of enzyme replacement therapy with recombinant enzymes.

221 Enzyme replacement therapy with recombinant human acid a

222 Enzyme replacement therapy with recombinant human alpha-

223 ance to iduronidase improved the efficacy of enzyme replacement therapy with recombinant iduronidase

224 utic options for NPC1 are few, and classical enzyme replacement therapy with the recombinant protein

225 We evaluated the effect of enzyme-replacement therapy with recombinant human alpha-

226 we evaluated the safety and effectiveness of enzyme-replacement therapy with sebelipase alfa (adminis