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.
Acer's pipeline includes three clinical-stage candidates: EDSIVO (celiprolol) for the treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; ACER-001 (a fully taste-masked, immediate release formulation of sodium phenylbutyrate) for the treatment of various inborn errors of metabolism, including
urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD) and osanetant for the treatment of induced Vasomotor Symptoms (iVMS) where Hormone Replacement Therapy (HRT) is likely contraindicated.
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.
- US-based synthetic biotic medicines developer Synlogic has dosed the first patient in its Phase 1b/2a clinical trial of SYNB1020, a synthetic biotic medicine being developed for the treatment of hyperammonemia, associated with cirrhosis and
urea cycle disorders,.
Clinical manifestations and growth of patients with
urea cycle disorders in Japan.
The website's focus was going to be about arginase deficiency, as well as
urea cycle disorders. It was going to be a patient advocacy website that would hopefully benefit current and new patients with overcoming the greatest hurdle of having arginase deficiency.
By mid-2017, Synlogic plans to initiate a Phase 1 healthy volunteers study for its lead candidate, SYNB1020, which is for the potential treatment of
Urea Cycle Disorders and hepatic encephalopathy, both diseases where patients experience elevated ammonia levels.
By mid-2017, Synlogic plans to initiate a Phase 1 healthy volunteers study for its lead candidate, SYNB1020, which is for the potential treatment of
Urea Cycle Disorders (UCD) and hepatic encephalopathy (HE), both diseases where patients experience elevated ammonia levels.
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.
Seminara, "A longitudinal study of
urea cycle disorders," Molecular Genetics and Metabolism, vol.
Urea cycle disorders (UCD) comprise at least eight conditions, caused by a deficiency of one of the essential proteins for the metabolism of nitrogen [1].
Gandolfo et al., "Sodium phenylbutyrate decreases plasma branched-chain amino acids in patients with
urea cycle disorders," Molecular Genetics and Metabolism, vol.
