The company's antibody - based product candidates target a number of potential indications including AL and AA
forms of amyloidosis, Parkinson's disease and other related synucleinopathies and novel cell adhesion targets involved in inflammatory diseases and cancers.
Not exact matches
The gene - silencing strategy, in the
form of RNA interference, has also showed promise against an uncommon neurodegenerative disease called hereditary ATTR
amyloidosis, as my colleague Sy Mukherjee wrote about in Fortune's year - end Investor's Guide.
Patisiran and the subcutaneously administered
form ALN - TTRsc02 are being developed for the treatment
of hereditary ATTR
amyloidosis.
The researchers also examined skin slices from two patients with another
form of nerve disease called AL
amyloidosis, caused by a bone marrow disorder.
Indeed, a series
of recent, exciting studies have shown that the pathologic hallmarks
of various PMDs, including Alzheimer's, Parkinson's, Huntington's diseases, and some
forms of systemic
amyloidosis, can be induced under experimental conditions by administration
of tissue homogenates carrying the respective misfolded proteins (Xing et al., 2001; Lundmark et al., 2002; Meyer - Luehmann et al., 2006; Clavaguera et al., 2009; Ren et al., 2009; Eisele et al., 2010; Grad et al., 2011; Luk et al., 2012; Morales et al., 2012; Mougenot et al., 2012; Stöhr et al., 2012; Iba et al., 2013).
The nature
of those plaques finally came into focus in 1984, when George Glenner, a research scientist at the University
of California, San Diego, identified the peptide called amyloid - beta and hypothesized that Alzheimer's was caused by «
amyloidosis»
of the brain, a process in which insoluble
forms of an amyloid protein accumulate.
While
amyloidosis may present itself as a symptom
of another medical condition, for those suffering from amyloid disease, the cause will be due to issues within the actual bone marrow whereby abnormal antibodies are produced
forming amyloid.