This guidance updates and replaces NICE's highly specialised technologies guidance 12 on cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2, which was available with managed access.
Neuronal ceroid lipofuscinosis type 1 (CLN1) disease, also known as infantile neuronal ceroid lipofuscinosis, is a rare and fatal neurodegenerative condition. CLN1 disease is caused by a deficiency in ...
StemCells, Inc. has received approval from the Institutional Review Board (IRB) of the Oregon Health & Science University (OHSU) to initiate a Phase I clinical trial of the company's proprietary human ...
Latus Bio Inc. has reported IND clearance by the FDA for LTS-101, a gene therapy candidate to treat the CNS manifestations of late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease. The ...
The National Institute for Health and Care Excellence (NICE) has rejected recommending cerliponase alfa (Brineura, BioMarin) for treating a rare form of Batten disease known as neuronal ceroid ...
Cerliponase alfa (Brineura, BioMarin) is indicated for 'the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) disease, also known as tripeptidyl peptidase 1 (TPP1) deficiency'. The list price ...
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