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Treatment with antiplatelet therapy does not reduce pain crises in children with sickle cell disease

Treatment with the antiplatelet agent prasugrel does not significantly reduce the rate of pain crises or severe lung complications in children with sickle cell disease, according to a report published in the New England Journal of Medicine describing one of the largest and most geographically diverse international clinical trials on sickle cell disease to date.

9 Dec 2015

Oxitec prepares to increase supplies of OX513A to address recent outbreak of Zika virus in Brazil

Intrexon Corporation, a leader in synthetic biology, today announced that Oxitec, a UK-based subsidiary of the Company, is prepared to increase supplies of its proprietary mosquito control solution, OX513A, through its Brazil facilities to assist Brazil in addressing the recent devastating outbreak of the Zika virus and other diseases transmitted by the Aedes aegypti mosquito.

8 Dec 2015

Children born to mothers with PCOS at increased risk of developing autism

Children born to mothers with polycystic ovarian syndrome, PCOS, are at an increased risk of developing autism spectrum disorders, according to a new epidemiological study from Sweden's Karolinska Institutet. The findings, which are published in the journal Molecular Psychiatry, support the notion that exposure to sex hormones early in life may be important for the development of autism in both sexes.

8 Dec 2015

Dompé’s investigational biotech molecule receives orphan drug designation for treatment of neurotrophic keratitis

The Dompé biopharmaceutical company announced today that the Committee for Orphan Medicinal Products of the European Medicines Agency (EMA) has officially designated recombinant human Nerve Growth Factor (rhNGF) - the investigational biotech molecule developed by Dompé based on research by Nobel Laureate Rita Levi Montalcini - as an orphan drug for the treatment of neurotrophic keratitis.

8 Dec 2015

Amgen’s Phase 2 data supports safety, efficacy of BLINCYTO in ALL patients with minimal residual disease

Amgen today announced that new data from three Phase 2 trials support the efficacy and safety of BLINCYTO (blinatumomab) in adults with acute lymphoblastic leukemia (ALL).

8 Dec 2015

Gene therapy shows promise in children with Wiskott-Aldrich syndrome

Researchers reported promising preliminary outcomes for the first four children enrolled in a U.S. gene therapy trial for Wiskott-Aldrich syndrome (WAS), a life-threatening genetic blood and immune disorder, at the 57th annual meeting of the American Society of Hematology (abstract #260).

7 Dec 2015

Age doesn't matter when considering HCT for patients with MDS, study reveals

Results from a prospective study of 1,280 patients with myelodysplastic syndrome (MDS) showed that survival at 100 days and at two years following hematopoietic cell transplant (HCT) for patients aged 65 and older is comparable to patients aged 55 to 64.

7 Dec 2015

Experimental gene therapy may improve health outcomes for patients with some forms of blood disorders

New research adds to a growing body of evidence that gene therapy, an experimental technique that involves correcting or replacing a person's mutated or malfunctioning genes, may improve health outcomes for patients with inherited bleeding and immune disorders as well as some forms of blood cancer.

7 Dec 2015

Infant death risk higher for mothers who gain weight gain between pregnancies

An analysis of pregnancies recorded on the Swedish Medical Birth Register indicates that the risk of a baby dying in its first year of life is greater among mothers who gain weight between their first and second pregnancies compared with women who do not gain weight.

3 Dec 2015

Lexicon announces top-line results from TELECAST Phase 3 study of telotristat etiprate

Lexicon Pharmaceuticals, Inc. announced today that top-line data from its TELECAST Phase 3 study showed results of telotristat etiprate in treating carcinoid syndrome in cancer patients with metastatic neuroendocrine tumors consistent with the clinical benefit observed in its pivotal TELESTAR study.

1 Dec 2015

TGen, Barrow researchers identify genetic risk factors linked to stress-induced cardiomyopathy

Researchers at the Translational Genomics Research Institute and Barrow Neurological Institute have for the first time identified genetic risk factors that are linked to stress-induced cardiomyopathy (SIC), a rare type of heart disease.

1 Dec 2015

Scientists can accurately measure microbial growth rates inside gastrointestinal tracts

For the first time, scientists can accurately measure population growth rates of the microbes that live inside mammalian gastrointestinal tracts, according to a new method reported in Nature Communications by a team at the Wyss Institute for Biologically Inspired Engineering at Harvard University and Harvard Medical School.

1 Dec 2015

Boston Scientific obtains FDA approval, CE Mark for AngioJet ZelanteDVT catheter to treat DVT

Boston Scientific has received United States (U.S.) Food and Drug Administration approval and CE Mark for the AngioJet ZelanteDVT thrombectomy catheter to treat deep vein thrombosis (DVT) in large-diameter upper and lower limb peripheral veins.

30 Nov 2015

Scientists discover genetic causes of Goldenhar syndrome

Some children are born with cleft palates and, of those children, some have an asymmetrical face and a malformed ear. A team of scientists led by Berlin-based researcher Enno Klußmann of the Max Delbrück Center for Molecular Medicine has taken an important step towards discovering the genetic causes of this condition, known as Goldenhar syndrome.

30 Nov 2015

Incidence of HIV in Europe reaches record high

Results of recent surveillance conducted by the European Centre for Disease Prevention and Control (ECDC) and the WHO Regional Office for Europe indicate that the annual number of newly diagnosed HIV infections in Europe reached an all-time high in 2014.

27 Nov 2015

Researchers use magic-angle-spinning NMR to unveil structure of CAP-Gly protein

A latticework of tiny tubes called microtubules gives your cells their shape and also acts like a railroad track that essential proteins travel on. But if there is a glitch in the connection between train and track, diseases can occur.

26 Nov 2015

Research reveals MECP2 Duplication Syndrome can be reversed

The methyl CpG binding protein 2 gene (MECP2) produces a protein of the same name, the level of which is critical for normal brain function. Mutations leading to protein under-expression cause Rett Syndrome while gene duplication causing over-expression lead to MECP2 Duplication Syndrome. Both disorders are severely debilitating childhood neurological diseases.

26 Nov 2015

Bile pigment may provide natural protection from heart attacks

New hope in the fight against cardiovascular disease has arrived, following breakthrough research identifying a pigment in our bile which could protect us.

26 Nov 2015

People with viral infections and identical gene mutations may be prone to hyperinflammatory disorder

A group of people with fatal H1N1 flu died after their viral infections triggered a deadly hyperinflammatory disorder in susceptible individuals with gene mutations linked to the overactive immune response, according to a study in The Journal of Infectious Diseases.

25 Nov 2015

People with chronic fatigue syndrome could experience higher levels of visual stress

People suffering from Chronic Fatigue Syndrome (CFS) could experience higher levels of visual stress than those without the condition, according to new research from the University of Leicester.

25 Nov 2015

Syndrome News and Research

Treatment with antiplatelet therapy does not reduce pain crises in children with sickle cell disease

Oxitec prepares to increase supplies of OX513A to address recent outbreak of Zika virus in Brazil

Children born to mothers with PCOS at increased risk of developing autism

Dompé’s investigational biotech molecule receives orphan drug designation for treatment of neurotrophic keratitis

Amgen’s Phase 2 data supports safety, efficacy of BLINCYTO in ALL patients with minimal residual disease

Gene therapy shows promise in children with Wiskott-Aldrich syndrome

Age doesn't matter when considering HCT for patients with MDS, study reveals

Experimental gene therapy may improve health outcomes for patients with some forms of blood disorders

Infant death risk higher for mothers who gain weight gain between pregnancies

Lexicon announces top-line results from TELECAST Phase 3 study of telotristat etiprate

TGen, Barrow researchers identify genetic risk factors linked to stress-induced cardiomyopathy

Scientists can accurately measure microbial growth rates inside gastrointestinal tracts

Boston Scientific obtains FDA approval, CE Mark for AngioJet ZelanteDVT catheter to treat DVT

Scientists discover genetic causes of Goldenhar syndrome

Incidence of HIV in Europe reaches record high

Researchers use magic-angle-spinning NMR to unveil structure of CAP-Gly protein

Research reveals MECP2 Duplication Syndrome can be reversed

Bile pigment may provide natural protection from heart attacks

People with viral infections and identical gene mutations may be prone to hyperinflammatory disorder

People with chronic fatigue syndrome could experience higher levels of visual stress

Bringing real-time analytics to downstream bioprocess monitoring

Designing fluidic architecture for scalable diagnostic platforms

Evaluating new approaches for rare disease genomics

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