Polaryx is developing patient-friendly oral small molecule therapeutics for Late Infantile Neuronal Ceroid Lipofuscinosis
(LINCL) and other forms of NCL, commonly known as Batten disease.
The abnormalities within the central nervous tissues were consistent with a diagnosis of neuronal ceroid lipofuscinosis
Neuronal ceroid lipofuscinosis
in the Czech Republic: analysis of 57 cases.
PPT1 and TPP1 activities in dried blood samples from patients with different forms of neuronal ceroid lipofuscinosis
and from healthy controls.
According to BioMarin, BMN 190 is a recombinant human tripeptidyl peptidase 1 (rhTPP1) for the treatment of patients with neuronal ceroid lipofuscinosis
type 2 (NCL-2), a form of Batten disease.
Association of mutations in a lysosomal protein with classical late-infantile neuronal ceroid lipofuscinosis
The request concerns Bio Marin's BMN-190, an investigational recombinant human tripeptidyl peptidase 1 for the treatment of Batten disease, or neuronal ceroid lipofuscinosis
type 2 (NCL-2).
The proposed Phase I trial is designed to investigate the safety of HuCNS-SC in 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.
Compound heterozygous genotype is associated with protracted juvenile neuronal ceroid lipofuscinosis
NASDAQ: STEM) today provided an update on the ongoing clinical development program of its proprietary HuCNS-SC([R]) product candidate (purified human neural stem cells) for neuronal ceroid lipofuscinosis
(NCL), often referred to as Batten disease.
s (NASDAQ: BMRN) Brineura (cerliponase alfa), the first treatment approved in the European Union for the treatment of neuronal ceroid lipofuscinosis
type 2, also known as tripeptidyl peptidase 1 deficiency, the company said.
5) a-Mannosidosis 5 4 (3-5) MLD (e) 32 5 (0-11) MPS I 18 4 (0-29) MPS II 24 3 (0-11) MPS IIIA 19 5 (1-17) MPS IIIB 16 6 (2-21) MPS IIIC 3 12 (6-20) MPS HID 3 2 (0-3) MPS IVA 16 7 (1-44) MPS VI 10 5 (0-16) Multiple sulphatase deficiency 2 7 (7) Neuronal ceroid lipofuscinosis
1 4 (infantile) (e) Niemann-Pick disease (A/B) (e) 9 26 (2-44) Niemann-Pick disease (C) (e) 10 13 (0-40) Pompe disease (e) 5 0.