The study analyzed that the lysosomal storage
disorder therapeutics pipeline comprises
approximately 74 drug candidates in different stages of development.
According to the research findings, most of the
drug candidates for lysosomal storage disorder pipeline are being developed to
be administered by intravenous route.
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Amicus Therapeutics, Inc. is using chaperone
advanced replacement therapy (CHART) technology platform for the development of
their drug candidates for the treatment of Fabry disease, a type of lysosomal
storage disorder. In a chaperone-advanced replacement therapy, a unique
pharmacological chaperone, binds to the infused therapeutic enzyme, stabilizing
the enzyme in its properly folded and active form. This proposed CHART
mechanism may allow for enhanced tissue uptake, greater lysosomal activity,
more reduction of storage material, and lower immunogenicity compared to ERT
alone.
The research also found that various companies use
natural sources for the development of lysosomal storage disorder therapeutics
pipeline. Protalix BioTherapeutics, Inc. is developing their drug candidates,
obtained from a natural source, for the treatment of Fabry disease.
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Some of the other key
players developing drugs for the treatment of lysosome storage disorder include
GlaxoSmithKline plc, Sanofi Genzyme, Amicus Therapeutics, Inc. and others.
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