The company is currently studying MGTA-456 in a Phase 2 clinical trial in patients older than 6 months of age with Hurler syndrome, cerebral adrenoleukodystrophy, metachromatic leukodystrophy or globoid
Clusters of expanded tubules are filled with large Sertoli cells with vacuolated, pale cytoplasm and internalized, globoid
deposits of basement membrane that project from thickened peritubular basement membrane (Figure 6, A and B).
In addition to MLD, this pattern is observed in single gene diseases that disrupt myelin tissue such as Pelizaeus-Merzbacher, globoid
cell leukodystrophy, and GM1 gangliosidosis (2,3).
The company plans to enroll 12 patients in the ongoing Phase 2 study in inherited metabolic disorders, which include cALD, Hurler syndrome, metachromatic leukodystrophy and globoid
The firm plans to enrol 12 patients in the ongoing Phase two study of inherited metabolic disorders including cALD, Hurler syndrome, metachromatic leukodystrophy and globoid
Developmental anatomy and immunocytochemistry reveal the neo-ontogenesis of the leaf tissues of Psidium myrtoides (Myrtaceae) towards the globoid
galls of Nothotrioza myrtoidis (Triozidae).
5), first segment as wide as long, second longer and wider than first; third, when present, narrower, shorter than second, and all approximately globoid
. Thorax: wing (Fig.
It is a native fruit of Brazil and can be found in the nature as shrubs or small trees, differing of the other Eugenia species because its leaves have primary venation adaxially concave, raceme with indefinite primary axis finished in vegetative branch, pyloric stem disc, hairy stylet and globoid
to subgloboid fruits (AMORIM; ALVES, 2012).
The gall shapes were characterized as globoid
, lenticular, marginal leaf roll, conical, cylindrical, fusiform, spherical and pocket shaped.
Gall morphotypes were classified in one of the following categories: clavate, conical, cylindrical, fusiform, globoid
, lenticular, rosette, bivalveshaped, hornshaped, leaf fold, marginal roll and pocket shaped, according to Isaias et al.
Orchard will also acquire rights to exclusively license three additional preclinical programmes from Telethon/Ospedale San Raffaele upon completion of clinical proof of concept studies for mucopolysaccharidosis type 1 (MPS1 or Hurler syndrome), chronic granulomatous disease (CGD) and globoid
cell leukodystrophy (GLD).
Krabbe disease (globoid
cell leukodystrophy) is an often-fatal lysosomal storage disease caused by the deficiency of the enzyme galactocerebrosidase (GALC; (8) EC 126.96.36.199), resulting in the loss of the myelin sheath in the central nervous system (1, 2).