Latest Lesch–Nyhan syndrome Stories

Teens with high levels of uric acid appear to be at increased risk for high blood pressure, according to results of research from scientists at Johns Hopkins Children’s Center. Although the findings do not establish a cause-and-effect link between uric acid and high blood pressure, they point to uric acid as one potential mechanism, or at least a biomarker, of disease, the researchers report in the April issue of the journal Hypertension. Analyzing more than 6,000 medical records of...

HAYWARD, Calif., Dec. 8, 2011 /PRNewswire/ -- Metabolex today announced positive results from its clinical study of arhalofenate in combination with febuxostat (Uloric(TM), Takeda Pharmaceutical Company Limited). A key goal in the treatment of gout is to address the patient's underlying hyperuricemia by treatment with drugs that lower serum uric acid (sUA). In the US, the goal of treatment is to reduce sUA levels to below 6 mg/dL. However, patients with severe tophaceous gout would...

HAYWARD, Calif., Nov. 1, 2011 /PRNewswire/ -- Metabolex, Inc., a biopharmaceutical company focused on the discovery and development of proprietary new medicines for the treatment of metabolic diseases, today announced that it will be presenting data on arhalofenate, a potential best-in-class treatment for gout, at the 2011 ACR/AHP Annual Scientific Meeting on Wednesday, November 9, 2011 in Chicago, IL. The presentation will describe the results of a pooled analysis of four Phase 2 studies...

HAYWARD, Calif., Oct. 20, 2011 /PRNewswire/ -- Metabolex, Inc., a biopharmaceutical company focused on the discovery and development of proprietary new medicines for the treatment of metabolic diseases, announced today that Dr. Raymond Urbanski has joined the company as Chief Medical Officer. Dr. Urbanski brings extensive clinical development experience to Metabolex, including expertise in all phases of clinical trial design and execution. Dr. Urbanski was the Vice President and Medical...

SAN DIEGO, Sept. 7, 2011 /PRNewswire/ -- Ardea Biosciences, Inc. (Nasdaq: RDEA) announced today that it has completed formal End-of-Phase 2 meetings with the U.S. Food and Drug Administration (FDA) and successfully reached agreement on the following key aspects of its proposed lesinurad Phase 3 development plan: the overall size and design of the planned Phase 3 clinical studies, the primary endpoints, the total safety database proposed for NDA filing, manufacturing plans for both drug...

As an alternative to a conventional treatment for gout that some patients may not respond to, patients with severe, chronic gout who received the medication pegloticase for 6 months had greater improvement in measures of uric acid levels as well as physical function and quality of life, according to a study in the August 17 issue of JAMA.Long-term urate (a salt derived from uric acid) lowering therapy in gout aims to maintain concentrations of uric acid (UA) below a certain level. However, it...

Injections of pegloticase, a modified porcine enzyme, can produce significant and sustained clinical improvements in 2 out of 5 patients with chronic gout that is resistant to conventional therapies, researchers report in the August 17, 2011, issue of JAMA.In two controlled clinical trials, pegloticase rapidly lowered high levels of uric acid, the biochemical abnormality in gout, and kept it in the normal range for six months or more in 42 percent of patients receiving the drug every two...

In humans, inherited mutations in a gene called HPRT1 lead to very specific self-destructive behavior. Boys with Lesch-Nyhan disease experience uncontrollable urges to bite their fingers, slam their arms into doorways and otherwise harm themselves.Puzzlingly, mice with mutations in the same gene don't behave differently than normal mice. Researchers at Emory University School of Medicine have identified a gene related to HPRT1, present in humans but not in mice that helps explain this...

LONDON, May 25, 2011 /PRNewswire/ -- Ardea Biosciences, Inc. (Nasdaq: RDEA) announced today that additional positive clinical and preclinical results from its development program of lesinurad, the Company's lead product candidate for the treatment of hyperuricemia and gout, were presented at the Annual European Congress of Rheumatology hosted by the European League Against Rheumatism (EULAR) in London, UK. Results from Primary Dosing Period and Ongoing Extension of Phase 2b Combination...

HAYWARD, Calif., May 19, 2011 /PRNewswire/ -- Metabolex, Inc., a biopharmaceutical company focused on the discovery and development of proprietary new medicines for the treatment of metabolic diseases, announced today that it has initiated a Phase 2 clinical trial of arhalofenate (MBX-102), its product candidate for the management of hyperuricemia in patients with gout. This randomized, double-blind, placebo-controlled study will evaluate the safety and urate-lowering activity of 400 and...