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

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

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

3 istopathological improvements observed after enzyme replacement therapy.

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

5 age accumulated gradually after cessation of enzyme replacement therapy.

6 exocrine pancreatic insufficiency requiring enzyme replacement therapy.

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

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

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

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

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

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

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

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

15 Although enzyme replacement therapy and bone marrow transplantati

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

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

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

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

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

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

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

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

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

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

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

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

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

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

30 Enzyme replacement therapy (ERT) and hematopoietic stem

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

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

33 Enzyme replacement therapy (ERT) effectively reverses st

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

35 Enzyme replacement therapy (ERT) for the lysosomal stora

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

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

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

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

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

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

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

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

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

45 Enzyme replacement therapy (ERT) is available for severa

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

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

48 Enzyme replacement therapy (ERT) partially and temporari

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

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

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

52 Enzyme replacement therapy (ERT) was eventually shown to

53 2 other options are available for ADA-SCID: enzyme replacement therapy (ERT) with pegylated bovine A

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

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

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

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

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

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

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

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

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

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

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

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

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

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

68 Enzyme replacement therapy for Gaucher disease is costly

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

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

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

72 Enzyme replacement therapy for lysosomal storage disease

73 Enzyme replacement therapy for lysosomal storage disorde

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

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

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

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

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

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

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

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

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

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

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

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

86 Enzyme replacement therapy has been used successfully in

87 Enzyme replacement therapy has been used successfully in

88 Enzyme replacement therapy has been used to treat mucopo

89 Enzyme replacement therapy has potential benefit but has

90 Enzyme replacement therapies have revolutionized patient

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

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

93 Enzyme replacement therapy in all males with Fabry disea

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

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

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

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

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

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

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

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

102 Enzyme replacement therapy is being developed for the la

103 Enzyme replacement therapy is currently available for th

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

105 Therefore, enzyme replacement therapy is not feasible using current

106 Enzyme replacement therapy is the principal treatment fo

107 Enzyme-replacement therapy is an established means of tr

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

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

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

111 Enzyme replacement therapy normalized the caloric densit

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

139 Enzyme replacement therapy with recombinant human alpha-

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

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

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