Intracerebroventricular Cerliponase Alfa for CLN2 Disease: Clinical Practice Considerations from US Clinics

被引：0
|
作者：
de los Reyes, E.
Lehwald, L.
Wang, R.
Augustine, E.
Berry-Kravis, E.
Butler, K.
Cormier, N.
Demarest, S.
Lu, S.
Madden, J.
Olaya, J.
See, S.
Vierhile, A.
Yang, A.
Leal-Pardinas, F.
机构：
来源：
ANNALS OF NEUROLOGY | 2019年 / 86卷
关键词：
Rare Diseases;
D O I：
暂无
中图分类号：
R74 [神经病学与精神病学];
学科分类号：
摘要：
205
引用
收藏
页码：S136 / S136
页数：1
相关论文
共 50 条
  • [21] Cerliponase alfa for the treatment of CLN2 disease in a patient cohort including children under 3 years of age
    Schulz, Angela
    Reyes, Emily C. de Los
    Specchio, Nicola
    Gissen, Paul
    Slasor, Peter
    Bondade, Shailesh
    Pfeffer, Jessica L. Cohen
    MOLECULAR GENETICS AND METABOLISM, 2024, 141 (02)
  • [22] CLN2 disease (neuronal ceroid lipofuscinosis type 2): Experience in the real world with cerliponase alfa intracerebroventricular enzyme replacement therapy in a public hospital in Cordoba, Argentina
    Seratti, Guillermo
    Munoz, Victor
    Guelbert, Norberto
    Jalil, Raul
    Velazquez, Daniel
    Pueyrredon, Francisco
    Guelbert, Guillermo
    Caraballo, Roberto
    Rodrigo, Tatiana
    MOLECULAR GENETICS AND METABOLISM, 2018, 123 (02) : S128 - S128
  • [23] 关于脑内灌注Cerliponase alfa治疗CLN2疾病的综述
    王丹
    孙婷婷
    孟雪莲
    陈长兰
    辽宁大学学报(自然科学版), 2019, 46 (03) : 237 - 243
  • [24] Exploring concurrent validity of the CLN2 Clinical Rating Scale: Comparison to PedsQL using cerliponase alfa clinical trial data
    Specchio, Nicola
    Gissen, Paul
    de los Reyes, Emily
    Olaye, Andrew
    Camp, Charlotte
    Curteis, Tristan
    Griffiths, Annabel
    Butt, Thomas
    Cohen-Pfeffer, Jessica
    Slasor, Peter
    Sisic, Zlatko
    Jain, Mohit
    Schulz, Angela
    PLOS ONE, 2024, 19 (05):
  • [25] Persistent Treatment Effect of Cerliponase Alfa in Children with CLN2 Disease: A 3 Year Update from an Ongoing Multicenter Extension Study
    Schulz, A.
    Specchio, N.
    Gissen, P.
    de los Reyes, E.
    Cahan, H.
    Slasor, P.
    Jacoby, D.
    Ajayi, T.
    ANNALS OF NEUROLOGY, 2019, 86 : S29 - S30
  • [26] Long-term Safety and Efficacy of Intracerebroventricular Enzyme Replacement Therapy with Cerliponase Alfa in Children with CLN2 Disease: Two Year Results from an Ongoing Multicenter Extension Study
    de Los Reyes, E.
    Schulz, A.
    Specchio, N.
    Gissen, P.
    Lehwald, L.
    Cahan, H.
    Slasor, P.
    Ajayi, T.
    Jacoby, D.
    ANNALS OF NEUROLOGY, 2018, 84 : S351 - S351
  • [27] Long-term safety and efficacy of intracerebroventricular enzyme replacement therapy with cerliponase alfa in children with CLN2 disease: interim results from an ongoing multicenter, multinational extension study
    Schulz, Angela
    Specchio, Nicola
    Gissen, Paul
    de los Reyes, Emily
    Cahan, Heather
    Slasor, Peter
    Ajayi, Temitayo
    Jacoby, David
    MOLECULAR GENETICS AND METABOLISM, 2017, 120 (1-2) : S120 - S120
  • [28] Long-term safety and efficacy of intracerebroventricular enzyme replacement therapy with cerliponase alfa in children with CLN2 disease: Two year results from an ongoing multicenter extension study
    Schulz, Angela
    Specchio, Nicola
    Gissen, Paul
    de los Reyesd, Emily
    Cahan, Heather
    Slasor, Peter
    Ajayi, Temitayo
    Jacoby, David
    MOLECULAR GENETICS AND METABOLISM, 2018, 123 (02) : S126 - S127
  • [29] Persistent treatment effect of cerliponase alfa in children with CLN2 disease: A 3 year update from an ongoing multicenter extension study
    Schulz, Angela
    Specchio, Nicola
    Gissen, Paul
    de los Reyes, Emily
    Cahan, Heather
    Slasor, Peter
    Jacoby, David
    Ajayi, Temitayo
    MOLECULAR GENETICS AND METABOLISM, 2019, 126 (02) : S133 - S133
  • [30] Long-term Safety and Efficacy of Intracerebroventricular Enzyme Replacement Therapy with Cerliponase Alfa in Children with CLN2 Disease: Interim Results from an Ongoing Multicenter, Multinational Extension Study
    de Los Reyes, E.
    Schulz, A.
    Specchio, N.
    Gissen, P.
    Cahan, H.
    Slasor, P.
    Ajayi, T.
    Jacoby, D.
    ANNALS OF NEUROLOGY, 2017, 82 : S336 - S336
12345