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Hemophilia is a rare, inherited bleeding disorder in which your blood doesn’t clot normally. If you have hemophilia, you may bleed for a longer time than others after an injury. You also may bleed internally, especially in your knees, ankles, and elbows. This bleeding can damage your organs or tissues and, sometimes, be fatal.
Baxter reports topline results from BAX 111 Phase 3 trial for treatment of von Willebrand disease

Baxter reports topline results from BAX 111 Phase 3 trial for treatment of von Willebrand disease

Baxter International Inc. today announced topline results from a Phase 3 clinical trial evaluating the safety, efficacy and pharmacokinetics (PK) of BAX 111. BAX 111 is a recombinant von Willebrand factor (rVWF) under investigation for the treatment of bleeding episodes in patients with von Willebrand disease, the most common type of inherited bleeding disorder. [More]
TSRI reveals new aspects of more potent anti-diabetic drugs with fewer serious side effects

TSRI reveals new aspects of more potent anti-diabetic drugs with fewer serious side effects

Scientists thought they basically knew how the most common drugs used to treat type 2 diabetes worked, but a new study from the Florida campus of The Scripps Research Institute (TSRI) reveals unexpected new aspects of the process. [More]
Scientists identify new biomarker linked to non-small cell lung cancer, head and neck cancers

Scientists identify new biomarker linked to non-small cell lung cancer, head and neck cancers

A team led by a scientist from the Florida campus of The Scripps Research Institute has identified a new biomarker linked to better outcomes of patients with head and neck cancers and non-small cell lung cancer. The work could help scientists develop new diagnostics and therapies and help physicians determine the best long-term treatments for patients with these cancers. [More]
New gene-editing system holds potential for treating many genetic disorders

New gene-editing system holds potential for treating many genetic disorders

Using a new gene-editing system based on bacterial proteins, MIT researchers have cured mice of a rare liver disorder caused by a single genetic mutation. [More]
Biogen Idec's ALPROLIX receives FDA approval for hemophilia B treatment

Biogen Idec's ALPROLIX receives FDA approval for hemophilia B treatment

Today Biogen Idec announced that the U.S. Food and Drug Administration has approved ALPROLIX [Coagulation Factor IX (Recombinant), Fc Fusion Protein], the first recombinant, DNA derived hemophilia B therapy with prolonged circulation in the body. [More]
TSRI scientists receive $2.3M to study viruses that cause tropical diseases

TSRI scientists receive $2.3M to study viruses that cause tropical diseases

The outbreak of dengue fever that infected some 20 people in Florida's Martin County late last year unnerved many who feared the tropical disease had once again established a foothold in Florida. The last outbreaks occurred in 2009 and 2010 in Key West—before that, the disease hadn't struck Florida in more than 70 years. [More]
Baxter plans to create two independent global healthcare companies

Baxter plans to create two independent global healthcare companies

Baxter International Inc. today announced plans to create two separate, independent global healthcare companies -- one focused on developing and marketing innovative biopharmaceuticals and the other on life-saving medical products. Both will be global leaders in their respective markets. [More]
Genable Technologies, Spark Therapeutics to advance novel therapy for rare form of retinitis pigmentosa

Genable Technologies, Spark Therapeutics to advance novel therapy for rare form of retinitis pigmentosa

Spark Therapeutics and Genable Technologies announced today that they have entered into a collaboration agreement for Genable's lead therapeutic to treat rhodopsin-linked autosomal dominant retinitis pigmentosa (RHO adRP), GT038. [More]
Biogen Idec's ALPROLIX gets Health Canada approval for hemophilia B

Biogen Idec's ALPROLIX gets Health Canada approval for hemophilia B

Today Biogen Idec announced that Health Canada has approved ALPROLIX [Coagulation Factor IX (Recombinant), Fc Fusion Protein], for the control and prevention of bleeding episodes and routine prophylaxis in adults, and children aged 12 and older, with hemophilia B. [More]
Scientists discover key mechanism that guides balance and limb movements

Scientists discover key mechanism that guides balance and limb movements

Scientists at The Scripps Research Institute (TSRI) have discovered an important mechanism underlying sensory feedback that guides balance and limb movements. [More]
Aradigm reports record revenue of $4.6M in fourth quarter 2013

Aradigm reports record revenue of $4.6M in fourth quarter 2013

Aradigm Corporation (the "Company") today announced financial results for the fourth quarter and full year ended December 31, 2013. [More]
Eisai collaborates with Biogen Idec to develop and commercialize treatment options for AD

Eisai collaborates with Biogen Idec to develop and commercialize treatment options for AD

Eisai Co., Ltd. and Biogen Idec announced today that they have entered into a collaboration to develop and commercialize two of Eisai's clinical candidates for Alzheimer's disease (AD), E2609 and BAN2401. [More]
LFB SA initiates Phase 3 program for human Factor VIIa in patients with congenital hemophilia

LFB SA initiates Phase 3 program for human Factor VIIa in patients with congenital hemophilia

LFB SA, through its rEVO Biologics subsidiary, announced today initiation of the global Phase 3 program for LR769, a novel recombinant form of human Factor VIIa, in patients with congenital hemophilia A or B with inhibitors. [More]
OPKO Health consolidated revenues increase about 30% to $20.7 million in Q4 2013

OPKO Health consolidated revenues increase about 30% to $20.7 million in Q4 2013

OPKO Health, Inc., a multi-national biopharmaceutical and diagnostics company, today reported operating and financial results for its 2013 fourth quarter and full year ended December 31, 2013. [More]
Bayer HealthCare joins NORD to promote awareness of Rare Disease Day

Bayer HealthCare joins NORD to promote awareness of Rare Disease Day

Bayer HealthCare is joining the National Organization for Rare Disorders (NORD) and others in raising awareness of Rare Disease Day, which is being celebrated today around the world. [More]
CSL Behring enrolls first patient in phase III study of C1-INH for prevention of HAE attacks

CSL Behring enrolls first patient in phase III study of C1-INH for prevention of HAE attacks

CSL Behring today announced it has enrolled the first patient in COMPACT, an international phase III study of a volume-reduced, subcutaneous formulation of C1-esterase inhibitor oncentrate in patients with frequent hereditary angioedema attacks (NCT01912456). [More]
TSRI scientists discover unusual protein that probably helps some bacteria evade immune response

TSRI scientists discover unusual protein that probably helps some bacteria evade immune response

A team led by scientists at The Scripps Research Institute (TSRI) has discovered an unusual bacterial protein that attaches to virtually any antibody and prevents it from binding to its target. Protein M, as it is called, probably helps some bacteria evade the immune response and establish long-term infections. [More]
TSRI scientists design artificial proteins as a candidate vaccine against RSV

TSRI scientists design artificial proteins as a candidate vaccine against RSV

Scientists at The Scripps Research Institute (TSRI) have invented a new method for designing artificial proteins, and have used it to make key ingredients for a candidate vaccine against a dangerous virus, respiratory syncytial virus (RSV), a significant cause of infant mortality. [More]
Biogen Idec, UCB sign agreements to commercialize treatment of MS and hemophilia in Asia

Biogen Idec, UCB sign agreements to commercialize treatment of MS and hemophilia in Asia

Biogen Idec and UCB announced today that they have signed exclusive agreements granting UCB the right to commercialize Biogen Idec products in South Korea, Hong Kong, Thailand, Singapore, Malaysia and Taiwan, and both develop and commercialize products in China. [More]
New data on extending the serum half-life of albumin in vivo announced by Novozymes Biopharma

New data on extending the serum half-life of albumin in vivo announced by Novozymes Biopharma

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