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The company's initial pipeline includes synthetic biotic medicines for the treatment of rare genetic diseases, such as urea cycle disorder and phenylketonuria.
Thankfully my parents found a wonderful organization called National Urea Cycle Disorder Foundation (NUCDF).
We are very pleased to enter this collaboration with Queen's University and look forward to working closely on this important program with Dr Funk who specialises in research related to urea cycle disorders.
With a portfolio of products for the treatment of Phenylketonuria, Gaucher Disease, Niemann Pick Type C, Hereditary Tyrosinemia Type 1, Urea Cycle Disorders and others, Dipharma SA works to provide solutions for people affected by inborn metabolic diseases at an affordable cost and with a global reach.
Ammonia is normally removed from circulation by incorporation into the urea cycle after condensation with bicarbonate and phosphorylation to form carbamoyl phosphate in the liver.
These range from orphan indications, such as rare inborn metabolic diseases including urea cycle disorders (UCD) to large indications such as acute-on-chronic liver failure (ACLF), and nonalcoholic steato-hepatits (NASH)/fibrosis.
The product, in a liquid formulation, helps eliminate excess ammonia that results from the accumulation of nitrogen in people with urea cycle disorders, which are inherited metabolic disorders.
Urea Cycle Disorders (UCD) are an important group of inborn errors of metabolism resulting from deficiencies of six different enzymes inherited as autosomal recessive, with the exception of Ornithine Transcarbamylase (OTC), which has X-linked inheritance.
We have started clinical experiments to test the effectiveness of this therapy in newborn children with inherited metabolic disorders, defect of liver metabolism and the first indication of urea cycle disorder.
This close cooperation will facilitate the treatment of children who suffer from urea cycle disorders, which is a genetic disorder caused by a deficiency of an enzymes in the urea cycle responsible for removing ammonia from the blood stream thereby cleaning it of toxins.