Primary Hyperoxaluria is a condition characterized by kidney and bladder stones that are recurrent. This condition further advances to end stage renal disease (ESRD), which is life – threatening since it prevents kidneys from filtering the fluids essential for the body from the waste products effectively.
This condition is a result of the overproduction of oxalate. Oxalate is excreted as a waste through urine after it gets filtered through the kidneys. This leads to abnormally high level of oxalate in urine which is why it is called as hyperoxaluria. When excreted, the oxalate can sometimes combine with calcium to form calcium oxalate, which is the main component of kidney and bladder stones. If these calcium oxalate compounds are deposited, it can lead to damage of kidneys or other organs as well. This will ultimately lead to blood in the urine and other urinary, kidney infections. This whole process weakens the kidneys and overtime, they lose the ability to excrete as much oxalate as they receive. The excess oxalate results in blood rise and these substances get deposited in tissues throughout the body, especially in bones and blood vessels. Bones are weakened and this leads to fractures.
Based on their severity and genetic reasons, primary hyperoxaluria are of three types. In Type 1, kidney stones appear right from childhood to early childhood. In Type 2, it develops later in life. In Type 3, affected individuals develop kidney stones in early childhood, but additional signs and symptoms are unclear since few cases have been reported so far. This condition is inherited in an autosomal recessive pattern, where both copies of gene in each cell have mutations. In cases caused genetically, the parents of the individual each carry one copy of the mutated gene but do not really show signs or symptoms of the condition.
Pipeline Review is an online portal that provides R&D new and online shop for reports about research & development in the biopharmaceutical industry. Ken Research’s Primary Hyperoxaluria-Pipeline Review, H2 2018 gives a snapshot of the therapeutic landscape of the disorder. The report covers therapeutic reviews based on their companies and research institutions based on their information derived from industry- specific sources. It provides descriptive drug profiles for the pipeline products which comprise product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities. The companies involved in therapeutic development in Primary Hyperoxaluria are Allena Pharmaceuticals Inc, Alnylam Pharmaceuticals Inc, Dicerna Pharmaceuticals Inc, Intellia Therapeutics Inc and OxThera AB. The report will help in procuring important information on competitors and their analysis to formulate R&D strategies to develop tactical initiatives in the target demographic.
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