Kaleido Biosciences announced data from a clinical study of KB195 in patients with urea cycle
disorders are being featured in a poster presentation during the Society for the Study of Inborn Errors of Metabolism Annual Symposium.
(Nasdaq: ACER), a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs, today announced it will host a conference call and webcast on Wednesday, July 31, 2019 at 11:00 am Eastern Time to discuss a detailed update on each of its pipeline programs, including ACER-001 for Urea Cycle
Disorders (UCDs) and Maple Syrup Urine Disease (MSUD), osanetant for neuroendocrine disorders, and EDSIVO for vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation.
Unbeknownst to everyone, Meegan suffered from Urea Cycle
Disorder, which meant having such a protein-heavy diet caused a build-up of ammonia in her blood.
N-carbamoylglutamate (NCG) is a feed additives obtained synthetically from sodium glutamate, which is a structural analogue of N-acetylglutamate , that is an essential factor of carbamoyl phosphate synthetase-1, a rate-liming enzyme that augments urea cycle
and the arginine (AGR) production process  and have favorable position of lower rumen breakdown compared to ARG .
ASA is a rare genetic urea cycle
disorder characterised by deficiency or lack of the enzyme argininosuccinate lyase (ASL), which is central to two metabolic pathways: the liver-based urea cycle
, which detoxifies ammonia and the citrulline-nitric oxide cycle, which synthesises nitric oxide from L-arginine.
The scope of the relationship includes R&D support for Promethera's HepaStem development program in acute on chronic liver failure (ACLF), non-alcoholic steatohepatitis (NASH) and urea cycle
disorder (UCD) in selected Asian markets.
The circular showed the expanded newborn screening test will be able to detect endocrine, amino and fatty acid disorders organic acid, urea cycle
defect, cystic fibrosis, hemoglobinopathies, biotinidase deficiency, and other disorders.
Currently, Sigmapharm's product line consists of Adefovir Dipivoxil Tablets, for hepatitis B, which is the first and only generic product equivalent to Hepsera tablets; Sodium Phenylbutyrate Powder, for urea cycle
disorders, which is equivalent to Buphenyl powder; Acitretin Capsules, equivalent to Soriatane capsules with a primary indication for the treatment of severe psoriasis; Dofetilide Capsules, equivalent to Tikosyn capsules for the treatment of arrhythmia; and Liothyronine Sodium Tablets, equivalent to Cytomel tablets for the treatment of hypothyroidism, which is now only available in 90-count (typical unit of use) bottles and is the most stable Liothyronine product on the market, with a recently Food and Drug Administration-approved shelf life of five years.
Zinc functions as antioxidant and is an important trace element that enhances the effectiveness of urea cycle
as well.5 The commonest site for the absorption of Zinc is jejunum, followed by the ileum and duodenum.
Ornithine transcarbamylase deficiency (OTC) is the most common urea cycle
disorder with an estimated prevalence of 1 per 80000 births.
Arginine supplementation in patients with CTLN1 drives the urea cycle
forward by replenishing ornithine and maximizing excretion of citrulline.
The urea cycle
and TCA are correlated and interdependent.