Devising effective enzyme replacement therapy for infantile onset neuronal ceroid lipofuscinosis (CLN1 disease)

2021 ◽  
Vol 132 (2) ◽  
pp. S28
Author(s):  
Jonathan D. Cooper ◽  
Ana C. Puhl ◽  
Sophie H. Wang ◽  
Elizabeth M. Eultgen ◽  
Keigo Takahashi ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Neuronal Ceroid Lipofuscinosis ◽  
Enzyme Replacement ◽  
Ceroid Lipofuscinosis

Review of Cerliponase Alfa: Recombinant Human Enzyme Replacement Therapy for Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2

2019 ◽  
Vol 35 (5) ◽  
pp. 348-353 ◽  
Author(s):  
Grace Lewis ◽  
Amanda M. Morrill ◽  
Stephanie L. Conway-Allen ◽  
Bernard Kim
Keyword(s):  
Adverse Events ◽  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Neuronal Ceroid Lipofuscinosis ◽  
Efficacy And Safety ◽  
Infantile Neuronal Ceroid Lipofuscinosis ◽  
Enzyme Replacement ◽  
Ceroid Lipofuscinosis ◽  
Tract Infections

The objective of this review is to summarize the pharmacology, efficacy, and safety of cerliponase alfa for the treatment of late infantile neuronal ceroid lipofuscinosis type 2 (CLN2). Cerliponase alfa is recombinant human tripeptidyl peptidase 1 enzyme replacement therapy. A phase 1/2 trial established the efficacy and safety of cerliponase alfa for treatment of neuronal ceroid lipofuscinosis type 2. Treatment with intracerebroventricular cerliponase alfa resulted in slower decline of motor and language functions compared with natural history controls. Common adverse events include convulsions, electrocardiography abnormalities, pyrexia, vomiting, and upper respiratory tract infections. Intracerebroventricular device–related adverse events also occur. Cerliponase alfa is the first therapy for neuronal ceroid lipofuscinosis type 2 that targets the disease etiology. Cerliponase alfa is effective in delaying the progression of motor language decline for patients with neuronal ceroid lipofuscinosis type 2.

scholarly journals Enzyme replacement therapy with recombinant pro-CTSD (cathepsin D) corrects defective proteolysis and autophagy in neuronal ceroid lipofuscinosis

Autophagy ◽  
2019 ◽  
Vol 16 (5) ◽  
pp. 811-825 ◽  
Author(s):  
André R. A. Marques ◽  
Alessandro Di Spiezio ◽  
Niklas Thießen ◽  
Lina Schmidt ◽  
Joachim Grötzinger ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Cathepsin D ◽  
Neuronal Ceroid Lipofuscinosis ◽  
Enzyme Replacement ◽  
Ceroid Lipofuscinosis
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