Hemophilia News and Research RSS Feed - Hemophilia News and Research

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.
Bayer, Dimension Therapeutics partner to develop new gene therapy for hemophilia A

Bayer, Dimension Therapeutics partner to develop new gene therapy for hemophilia A

Dimension Therapeutics, a company focused on developing novel adeno-associated virus (AAV) gene therapy treatments for rare diseases, today announced it has entered into a collaboration with Bayer HealthCare (Bayer) for the development and commercialization of a novel gene therapy for the treatment of hemophilia A. [More]
Fragile Y hypothesis explains the factors behind chromosome loss

Fragile Y hypothesis explains the factors behind chromosome loss

A UT Arlington research team says their study of genetic information from more than 4,000 beetle species has yielded a new theory about why some species lose their Y chromosome and others, such as humans, hang on to it. [More]
Leading experts from key sectors discuss challenges in 340B Drug Discount Program

Leading experts from key sectors discuss challenges in 340B Drug Discount Program

Leading health care policy experts from community health centers, patient and clinician specialty groups, industry organizations and academic institutions convened yesterday to discuss challenges in the federal 340B Drug Discount Program, and the importance of preserving aspects that truly serve vulnerable, uninsured and underinsured patients. [More]
Alnylam announces new pre-clinical results with DC for ALN-CC5 to treat complement-mediated diseases

Alnylam announces new pre-clinical results with DC for ALN-CC5 to treat complement-mediated diseases

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today new pre-clinical results with its Development Candidate (DC) for ALN-CC5, a subcutaneously administered investigational RNAi therapeutic targeting complement component C5 in development for the treatment of complement-mediated diseases. [More]
TSRI develops accurate structural map of complex molecular machines in cells

TSRI develops accurate structural map of complex molecular machines in cells

A team led by researchers at The Scripps Research Institute (TSRI) has used advanced electron microscopy techniques to determine the first accurate structural map of Mediator, one of the largest and most complex "molecular machines" in cells. [More]
Nektar reports etirinotecan pegol Phase 2 study results in patients with Avastin-refractory high-grade glioma

Nektar reports etirinotecan pegol Phase 2 study results in patients with Avastin-refractory high-grade glioma

Nektar Therapeutics announced today new data from an investigator-sponsored Phase 2 study of NKTR-102 (etirinotecan pegol) in patients with Avastin-refractory high-grade glioma conducted at Stanford Cancer Institute under the direction of Lawrence Recht, M.D., Professor of Neurology and Neurosurgery, with co-investigator Seema Nagpal, M.D., Clinical Assistant Professor of Neurology and Neurological Sciences, Stanford School of Medicine. [More]
CIRM grants $5.6 million to develop HIV/AIDS therapeutic using Sangamo's ZFN genome-editing technology

CIRM grants $5.6 million to develop HIV/AIDS therapeutic using Sangamo's ZFN genome-editing technology

Sangamo BioSciences, Inc. announced that the California Institute for Regenerative Medicine (CIRM) has granted a $5.6 million Strategic Partnership Award to fund clinical studies at City of Hope to develop a potentially curative ZFP Therapeutic for HIV/AIDS based on the application of Sangamo's zinc finger nuclease (ZFN) genome-editing technology in hematopoietic stem/progenitor cells (HSPCs). [More]
TPP wins $500,000 grant from Biogen Idec to help students start and run own businesses

TPP wins $500,000 grant from Biogen Idec to help students start and run own businesses

The Biogen Idec Foundation has awarded a $500,000 grant to The Possible Project (TPP), a non-profit organization that teaches high school students how to start and run their own businesses through a tailored curriculum, and access to technology and to mentors from the local business community. [More]
Half-life extension technology: an interview with Dermot Pearson, Novozymes Biopharma

Half-life extension technology: an interview with Dermot Pearson, Novozymes Biopharma

A drug's half-life indicates how long a substance will remain active in the body. It is essentially the period of time that it takes for the concentration of the amount of the drug to be reduced by half, and therefore, determines how frequently a drug needs to be administered to maintain its therapeutic effect. [More]
TSRI researchers discover key proteins involved in one type of DNA repair gone awry

TSRI researchers discover key proteins involved in one type of DNA repair gone awry

Accumulation of DNA damage can cause aggressive forms of cancer and accelerated aging, so the body's DNA repair mechanisms are normally key to good health. However, in some diseases the DNA repair machinery can become harmful. [More]
Researchers discover key proteins involved in DNA repair gone awry

Researchers discover key proteins involved in DNA repair gone awry

Accumulation of DNA damage can cause aggressive forms of cancer and accelerated aging, so the body's DNA repair mechanisms are normally key to good health. However, in some diseases the DNA repair machinery can become harmful. [More]
Study led by TSRI scientists likely to influence future designs for HIV vaccines, antibody-based therapies

Study led by TSRI scientists likely to influence future designs for HIV vaccines, antibody-based therapies

A vaccine or other therapy directed at a single site on a surface protein of HIV could in principle neutralize nearly all strains of the virus—thanks to the diversity of targets the site presents to the human immune system. [More]
Scripps Research Institute scientists show AIDS vaccine could work against changeable site on HIV

Scripps Research Institute scientists show AIDS vaccine could work against changeable site on HIV

A vaccine or other therapy directed at a single site on a surface protein of HIV could in principle neutralize nearly all strains of the virus-thanks to the diversity of targets the site presents to the human immune system. [More]
FDA approves Bayer's Kogenate FS antihemophilic factor VIII to prevent bleeding episodes in adults with hemophilia A

FDA approves Bayer's Kogenate FS antihemophilic factor VIII to prevent bleeding episodes in adults with hemophilia A

Bayer HealthCare announced today that the U.S. Food and Drug Administration (FDA) has approved a new indication for Bayer's Kogenate® FS antihemophilic factor VIII (recombinant), for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults with hemophilia A. [More]
Voluntis closes €20.75 million ($29 million) Series D financing round

Voluntis closes €20.75 million ($29 million) Series D financing round

Voluntis, a company pioneering therapeutic companion software, today announced the completion of its Series D financing round. With a total of €20.75 million ($29 million), this fund raising is one of the largest rounds of investment in the m-health sector this year. [More]
TSRI scientists discover new HIV target

TSRI scientists discover new HIV target

A team led by scientists at The Scripps Research Institute (TSRI) working with the International AIDS Vaccine Initiative (IAVI) has discovered a new vulnerable site on the HIV virus. The newly identified site can be attacked by human antibodies in a way that neutralizes the infectivity of a wide variety of HIV strains. [More]
Biogen Idec's revenue increases 51% to $2.1 billion in first quarter 2014

Biogen Idec's revenue increases 51% to $2.1 billion in first quarter 2014

Biogen Idec Inc. today reported first quarter 2014 results, including revenue of $2.1 billion, a 51% increase compared to the first quarter of 2013. [More]
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]