The company's initial pipeline includes synthetic biotic medicines for the treatment of rare genetic diseases, such as urea cycle disorder
In addition, PRX-ASL is reportedly the second enzyme replacement therapy to receive orphan drug designation for a Urea Cycle Disorder
, added the company.
Thankfully my parents found a wonderful organization called National Urea Cycle Disorder
Keywords: Newborn, urea cycle disorder
, peritoneal dialysis
In this case, the patient presented with vague symptoms and a critical (>187 [micro]g/dL or >110 [micro]mol/L) plasma ammonia which raised suspicion for a urea cycle disorder
HepaStem treatment has been shown to be safe in a phase 1/2 study in 20 urea cycle disorder
A doctor from the medical team said that the boy was suffering from a urea cycle disorder
, or a genetic disorder caused by a mutation that results in a deficiency of one of the six enzymes in the urea cycle.
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
OTC deficiency is the only X-linked urea cycle disorder
Hyperammonemia is highly suggestive of a urea cycle disorder
A urea cycle disorder
is one of six genetic disorders causing an enzyme deficiency in the urea cycle.
Acer said it also intends to use the financing to support manufacturing and clinical development of ACER-001, a proprietary taste-masked, immediate-release formulation of sodium phenylbutyrate (NaPBA) for Maple Syrup Urine Disease (MSUD) and Urea Cycle Disorder
(UCD), serious genetic diseases which impact amino acid and ammonia metabolism, respectively.