NCL

(redirected from Neuronal ceroid lipofuscinosis)
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NCL

GOST 7.67 Latin three-letter geocode for New Caledonia. The code is used for transactions to and from New Caledonian bank accounts and for international shipping to New Caledonia. As with all GOST 7.67 codes, it is used primarily in Cyrillic alphabets.
References in periodicals archive ?
MRI of neuronal ceroid lipofuscinosis. Part I: cranial MRI of 30 patients with juvenile neuronal ceroid lipofuscinosis.
The abnormalities within the central nervous tissues were consistent with a diagnosis of neuronal ceroid lipofuscinosis (NCL).
Diagnostic value of electron microscopy in a case of juvenile neuronal ceroid lipofuscinosis. Ultrastruct Pathol 2001; 25: 285-8.
Detection of eight novel palmitoyl protein thicesterase (PPT) mutations underlying infantile neuronal ceroid lipofuscinosis (INCL; CLN1).
Late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, a form of Batten disease, is characterized by seizures, rapid deterioration of language and motor functions, cognitive decline, loss of vision and blindness, and premature death by mid-childhood.
a leading clinical-stage biopharmaceutical company focused on developing novel gene and cell therapies for life-threatening rare diseases, and REGENXBIO, a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAV Technology Platform, today announced a license agreement to REGENXBIOs NAV AAV9 vector for the treatment of four diseases: Sanfilippo syndrome type A (MPS IIIA), Sanfilippo syndrome type B (MPS IIIB), Infantile Batten Disease, also known as neuronal ceroid lipofuscinosis type 1 (CLN1 Disease), and Juvenile Batten Disease, also known as neuronal ceroid lipofuscinosis type 3 (CLN3 Disease).
Regenxbio is developing a new product candidate, RGX-181, for the treatment of late-infantile neuronal ceroid lipofuscinosis type 2 disease, one of the most common forms of Batten disease caused by mutations in the tripeptidyl peptidase 1 gene.
(STEM) has discontinued its Phase lb clinical trial in neuronal ceroid lipofuscinosis (NCL, also referred to as Batten disease), a rare and fatal neurodegenerative disorder in children, due to a lack of patients who met the criteria for participation.
The proposed Phase 1 trial is designed to evaluate the safety and preliminary efficacy of HuCNS-SC for the treatment of infantile and late-infantile neuronal ceroid lipofuscinosis (NCL), the most severe forms of a group of disorders commonly referred to as Batten disease.
Mutations in the palmitoyl protein thicesterase gene causing infantile neuronal ceroid lipofuscinosis. Nature 1995;376:584-7.
Abeona Therapeutics announced that the European Medicines Agency Committee for Orphan Medicinal Products has granted Orphan Drug Designation for Abeona's gene therapy program ABO-202 for the treatment of subjects with neuronal ceroid lipofuscinosis, also known as Batten Disease, a fatal lysosomal storage disease that primarily affects the nervous system in children.
M2 PHARMA-March 2, 2018-EMA Grants Polaryx Therapeutics Orphan Drug Designation for PLX-200 for Treatment of Neuronal Ceroid Lipofuscinosis
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