Amicus Therapeutics (FOLD: Quote) has entered into a collaboration with Biogen Idec (BIIB) to discover, develop and commercialize novel small molecules for the treatment of Parkinson's disease. The collaboration will build upon preclinical studies at Amicus and independent published research that suggest increasing activity of the lysosomal enzyme glucocerobrosidase (GCase) in the brain may correct alpha-synuclein pathology and other deficits associated with Parkinson's disease. FOLD closed Tuesday's trading at $2.66, up 4.31%. Conatus Pharmaceuticals Inc. (CNAT: Quote), which made its stock market debut as recently as July of this year, has initiated a phase II study of Emricasan in patients with severe alcoholic hepatitis. This study is being conducted by the Translational Research and Evolving Alcoholic Hepatitis Treatment (TREAT) Consortium consisting of the Mayo Clinic Rochester, Indiana University, and Virginia Commonwealth University in collaboration with the National Institute on Alcohol Abuse and Alcoholism. CNTA closed Tuesday's trading 0.47% higher at $8.62. Exelixis Inc. (EXEL: Quote) has initiated a phase III trial, dubbed CELESTIAL, comparing Cabozantinib with placebo in patients with advanced hepatocellular carcinoma who have previously been treated with Sorafenib. The primary endpoint for the trial is overall survival. EXEL touched a new 52-week high of $5.64 on Tuesday before … Continue reading →

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MONDAY, Sept. 9 (HealthDay News) -- A genetic deletion may be linked to some cases of early onset Parkinson's disease, researchers say. The investigators found that people aged 35 to 64 who were missing DNA on a specific part of chromosome 22 were about 90 times more likely to develop Parkinson's than people from the same age group in the general population. People with this inherited genetic condition -- called 22q11.2 deletion syndrome -- have about 50 genes missing on chromosome 22. The condition occurs in about one in 2,000 to 4,000 people, and those with this genetic deletion may have birth defects (including heart defects), learning or speech difficulties, anxiety disorders, or schizophrenia. Previously reported cases of patients with 22q11.2 deletion syndrome and Parkinson's disease symptoms have indicated that there may be a link between the two conditions, according to the researchers from the Center for Addiction and Mental Health and University Health Network in Toronto. Dr. Anne Bassett, director of the Center for Addiction and Mental Health's Clinical Genetics Research Program, said 22q11.2 deletion syndromes has been studied in children and adolescents, but less is known about the syndrome in adults. In addition, in some cases, Parkinson's disease … Continue reading →

CRANBURY, N.J., Sept. 10, 2013 (GLOBE NEWSWIRE) -- Amicus Therapeutics (FOLD), a biopharmaceutical company at the forefront of therapies for rare and orphan diseases, has entered a collaboration with Biogen Idec (BIIB) to discover, develop and commercialize novel small molecules for the treatment of Parkinson's disease. The collaboration will build upon preclinical studies at Amicus and independent published research that suggest increasing activity of the lysosomal enzyme glucocerobrosidase (GCase) in the brain may correct alpha-synuclein pathology and other deficits associated with Parkinson's disease. "Our collaboration with Amicus complements our current strategy to identify and develop novel therapies to address Parkinson's disease," said Tim Harris, Senior Vice President of Translational Medicine at Biogen Idec. "Amicus has been a pioneer in the discovery of novel small molecules that increase GCase activity in the brain, and we look forward to working together to discover potential treatments for Parkinson's disease." Under terms of the multi-year agreement, Amicus and Biogen Idec will collaborate in the discovery of a new class of small molecules that target the GCase enzyme, for further development and commercialization by Biogen Idec. Biogen Idec will be responsible for funding all discovery, development, and commercialization activities. In addition Amicus will be reimbursed … Continue reading →

BOSTON, Sept. 10, 2013 /PRNewswire/ -- Rhythm announced today that The Michael J. Fox Foundation for Parkinson's Research (MJFF) has awarded a $1.35 million research grant to Rhythm to help fund a Phase 2 clinical trial of RM-131, the company's novel ghrelin agonist, for the treatment of refractory constipation in patients with Parkinson's disease (PD). This study expands the ongoing Phase 2 clinical program for RM-131 for the treatment of both upper and lower gastrointestinal (GI) functional disorders. "As we work toward a cure for Parkinson's, alleviating the symptoms of disease is our best approach to improve quality of life for the millions living with PD. Development of a treatment for painful and debilitating constipation will have significant meaning to this population," said Maurizio Facheris, MD, MJFF associate director of research programs. Constipation is common among people with Parkinson's, with studies reporting more than 50% of PD patients suffering from moderate to severe constipation. Many of these patients are refractory to existing therapy. In addition, GI dysfunction in PD can affect the upper GI tract, resulting in gastroparesis. Overall, the symptoms of upper and lower GI functional disorders can be debilitating and disruptive to the lives of PD patients and, … Continue reading →