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Eli Lilly receives FDA complete response letter for liprotamase NDA

Eli Lilly and Company has received a complete response letter from the U.S. Food and Drug Administration for its New Drug Application for liprotamase, a non-porcine pancreatic enzyme replacement therapy, under investigation for the treatment of exocrine pancreatic insufficiency.

16 Apr 2011

Synageva BioPharma closes $25 million private equity financing

Synageva BioPharma Corp., a privately held biopharmaceutical company developing therapeutic products for rare disorders, announced today the closing of a $25 million private equity financing.

21 Mar 2011

Synageva supports Fourth International Rare Disease Day

Synageva BioPharma Corp., a privately held biopharmaceutical company that is developing therapeutic products for rare disorders, announced today that the company is supporting the Fourth International Rare Disease Day observance on February 28, 2011.

28 Feb 2011

AMT 2010 total net income increases to EUR 1.4 million

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, today reported its results for the year to December 31, 2010.

16 Feb 2011

Women with PCOS face high risk of type 2 diabetes due to aberrations in fat tissue

A study from the University of Gothenburg, shows that women with polycystic ovary syndrome (PCOS) have aberrations in their adipose tissue.

17 Jan 2011

AMT enters agreement with Consortium for clinical development of novel gene therapy to treat Sanfilippo B

Amsterdam Molecular Therapeutics, a leader in human gene therapy, announced today that it has entered into an agreement with Institut Pasteur, Paris, France, and a group of French research institutes (together the "Consortium") to support clinical development of a novel gene therapy to treat Sanfilippo B.

10 Jan 2011

New mouse model with deficiency of lipoprotein lipase in neurons

Researchers at the University of Colorado School of Medicine have created a new and exciting mouse model to study how lipid sensing and metabolism in the brain relate to the regulation of energy balance and body weight. The research team, led by Hong Wang, PhD, created mice with a deficiency of lipoprotein lipase (LPL) in neurons, and observed two important reactions.

5 Jan 2011

New drug appears to help patients with chronic myeloid leukemia

A new drug appears to help chronic myeloid leukemia patients who are out of treatment options after first- and second-line drugs have failed them or because their cancer cells have a mutation that makes them resistant from the start, researchers reported at the 52nd Annual Meeting of the American Society of Hematology.

7 Dec 2010

$Updated Phase 1 study data of ponatinib in patients with refractory CML, Ph+ ALL presented at ASH$

Updated Phase 1 study data of ponatinib in patients with refractory CML, Ph+ ALL presented at ASH

ARIAD Pharmaceuticals, Inc. today announced updated clinical data from a fully enrolled and ongoing Phase 1 study of its investigational pan-BCR-ABL inhibitor, ponatinib, in patients with resistant and refractory chronic myeloid leukemia and Philadelphia-positive acute lymphoblastic leukemia.

6 Dec 2010

Angptl4 protein deficiency could promote inflammatory response in people who consume high saturated fat

A new report in the December Cell Metabolism identifies a protein without which diets high in saturated fat lead to a massive inflammatory response that can prove fatal. The studies in mice suggest that deficiencies in this protective pathway could promote inflammation in those who regularly consume high levels of saturated fat.

1 Dec 2010

Synageva's SBC-102 enzyme replacement therapy for LAL deficiency granted European orphan product designation

Synageva BioPharma Corp., a privately held biopharmaceutical company, announced that the European Medicines Agency (EMA) granted orphan product designation for SBC-102 (Recombinant Human Lysosomal Acid Lipase), the Company's enzyme replacement therapy in development to treat Lysosomal Acid Lipase (LAL) Deficiency.

10 Nov 2010

Synageva receives $2.3 million under QTDP program

Synageva BioPharma Corp., a privately held biopharmaceutical company developing novel treatments for rare diseases, today announced that the US Treasury Department awarded approximately $2.3 million to Synageva BioPharma as part of the Qualifying Therapeutic Discovery Project Program.

4 Nov 2010

Injecting apoA-V protein may reduce high triglyceride levels: Research

Injecting a protein that helps break down triglycerides may someday help treat an inherited form of high triglycerides, according to a new study in Arteriosclerosis, Thrombosis, and Vascular Biology, an American Heart Association journal.

22 Oct 2010

Progenika receives CE Mark approval to market LPLchip in Europe

Progenika Biopharma S.A., a pioneer in the field of personalized medicine, announced today that it has obtained the CE Mark allowing the sale in Europe of its LPLchip(R), the first DNA chip to detect mutations in the lipoprotein lipase (LPL) gene.

27 Sep 2010

Amsterdam Molecular first half 2010 net loss in line with last year

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, today reported its results for the first half year of 2010.

31 Aug 2010

Continuous boosting of natural painkiller shuts down brain cell receptor response

ERepeatedly boosting brain levels of one natural painkiller soon shuts down the brain cell receptors that respond to it, so that the painkilling effect is lost, according to a surprising new study led by Scripps Research Institute and Virginia Commonwealth University scientists. The study has important implications for drug development.

25 Aug 2010

AMT's MAA for LPLD gene therapy product Glybera on schedule

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, today announced that the Marketing Authorisation Application (MAA) for Glybera(R) remains on schedule following meetings with the European Medicine Agency (EMA) concerning the Day 120 List of Questions. The company is confident that Glybera(R), a gene therapy product for lipoprotein lipase deficiency (LPLD), remains on track for a regulatory decision by mid-2011.

18 Aug 2010

YM concludes dose-escalation in CYT387 Phase I/II clinical trial for myelofibrosis

YM BioSciences Inc., announced the conclusion of dose-escalation in the Phase I portion of its Phase I/II clinical trial of CYT387 at Mayo Clinic in patients with myelofibrosis. In total, 21 patients were treated in Phase I, with no voluntary withdrawals reported. CYT387 has shown significant activity in reducing spleen size and controlling constitutional symptoms in these patients.

5 Aug 2010

Survey links autism with digestive problems, intestinal inflammation

Autism is the fastest growing developmental disorder in the world. With 1 child in 91 facing the disorder, the diagnosis is more common than pediatric cancer, diabetes, and AIDS combined.

22 Jul 2010

New findings show why some people suffer from high levels of triglycerides

New findings reported in the July issue of Cell Metabolism, a Cell Press publication, are offering new leads as to why some people might suffer from high levels of triglycerides. High triglycerides are a risk factor for atherosclerosis and cardiovascular disease. They can also lead to inflammation of the pancreas, the researchers said.

8 Jul 2010