Urea
cycle disorders (UCDs) are an inborn error of metabolism, that occurs due to the deficiencies
in any of the six enzymes and two transport proteins involved in urea
biosynthesis. These disorders cause inborn errors in urea synthesis that lead
to an accumulation of ammonia in blood and brain.
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UCDs
clinically present as recurrent episodes of hyperammonemia manifested by
vomiting, lethargy and coma. The diagnosis of UCDs occurs during neonatal
screening; while, some milder cases can diagnose later. The management of UCDs
is based on minimizing the nitrogen load by decreasing dietary protein intake.
Thus, patients generally consume a protein-restricted diet in order to minimize
the production of ammonia from dietary nitrogen.
The drug
candidates in UCDs therapeutics pipeline include, but not limited to, SYNB1020
and mRNA therapeutics. Some of the major companies having pipeline drugs for
UCDs includes PhaseRx Inc., Synlogic and Castle Creek Pharma.
The report
provides a comprehensive understanding of the pipeline activities covering all
drug candidates under various stages of development, with the detailed analysis
of pipeline and clinical trials.
Pipeline
analysis of drugs by phases includes product description and development
activities including information about clinical results, designations,
collaborations, licensing, grants, technology, and others.
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