Overall, the results observed would not have been anticipated in the absence of treatment. The effect of vestronidase alfa on the central nervous system manifestations of MPS VII has not been determined.The FDA approved vestronidase alfa-vjbk based primarily on evidence from one clinical trial (NCT02230566) of 12 participants with mucopolysaccharidosis VII. The trial was conducted at four sites in the United States.The benefit and side effects of vestronidase alfa were based primarily on one trial. Participants were randomly assigned to four groups. Three groups of participants received placebo treatment before starting vestronidase alfa treatment and one group received vestronidase alfa only. vestronidase alfa or placebo were given once every two weeks as intravenous (IV) infusions. Neither participants nor healthcare providers knew which treatment was given until after the trial was competed.The benefit of 24 weeks of vestronidase alfa treatment was primarily evaluated by the 6-minute walking test (6MWT) and compared to placebo treatment in ten participants who could perform the test. The 6MWT measured the distance a patient could walk on a flat surface in 6 minutes. An additional follow-up using 6MWT was done for up to 120 weeks.The application for vestronidase alfa was granted fast track designation, orphan drug designation, and a rare pediatric disease priority review voucher. This was the twelfth rare pediatric disease priority review voucher issued.The U.S. Food and Drug Administration (FDA) granted approval of Mepsevii to Ultragenyx Pharmaceutical, Inc, and required the manufacturer to conduct a post-marketing study to evaluate the long-term safety of the product. References
External links
"Vestronidase alfa".
The misfolded nature of protein aggregates causes a multitude of aberrant interactions with a multitude of cellular components, including membranes, protein receptors, soluble proteins, RNAs, small metabolites, etc. Histological staining
In the clinical setting, amyloid diseases are typically identified by a change in the spectroscopic properties of planar aromatic dyes such as thioflavin T, congo red or NIAD-4. In general, this is attributed to the environmental change, as these dyes intercalate between beta-strands to confine their structure.Congo Red positivity remains the gold standard for diagnosis of amyloidosis. In general, binding of Congo Red to amyloid plaques produces a typical apple-green birefringence when viewed under cross-polarized light. Recently, significant enhancement of fluorescence quantum yield of NIAD-4 was exploited to super-resolution fluorescence imaging of amyloid fibrils and oligomers. To avoid nonspecific staining, other histology stains, such as the hematoxylin and eosin stain, are used to quench the dyes activity in other places such as the nucleus, where the dye might bind. Modern antibody technology and immunohistochemistry has made specific staining easier, but often this can cause trouble because epitopes can be concealed in the amyloid fold; in general, an amyloid protein structure is a different conformation from the one that the antibody recognizes. See also
JUNQ and IPOD
Proteopathy
Protein aggregation predictors

References
External links

Bacterial Inclusion Bodies Contain Amyloid-Like Structure at SciVee
Amyloid Cascade Hypothesis
Amyloid: Journal of Protein Folding Disorders web page
Role of anesthetics in Alzheimers disease: Molecular details revealed