Hemophilia News and Research

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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.

Study brings effective hepatitis C cure closer to reality

Scientists at The Scripps Research Institute have determined the most detailed picture yet of a crucial part of the hepatitis C virus, which the virus uses to infect liver cells. The new data reveal unexpected structural features of this protein and should greatly speed efforts to make an effective hepatitis C vaccine.

29 Nov 2013

Human Treg responses allow sustained recombinant adeno-associated virus-mediated transgene expression

Successful gene therapy is based on the effective delivery and maintained expression of healthy copies of a gene into tissues of individuals with a disease-associated genetic mutation.

16 Nov 2013

Generic drug gabapentin appears to be safe, effective in treating alcohol dependence

The generic drug gabapentin, which is already widely prescribed for epilepsy and some kinds of pain, appears to be safe and effective in the treatment of alcohol dependence. The finding comes from a 150-patient randomized, placebo-controlled, double blind clinical trial conducted by scientists at The Scripps Research Institute.

4 Nov 2013

Researchers overcome obstacle to using viruses to deliver therapeutic genes

Scientists in The Research Institute at Nationwide Children's Hospital have found a way to overcome one of the biggest obstacles to using viruses to deliver therapeutic genes: how to keep the immune system from neutralizing the virus before it can deliver its genetic payload. In a study published recently in Molecular Therapy, researchers found that giving subjects a treatment to temporarily rid the body of antibodies provides the virus safe passage to targeted cells, allowing it to release a corrective or replacement gene to treat disease.

30 Oct 2013

Biogen Idec Q3 total revenues increase 32% to $1.8 billion

Biogen Idec Inc. today reported third quarter 2013 total revenues of $1.8 billion, an increase of 32% over the third quarter of 2012. Non-GAAP diluted EPS for the third quarter of 2013 were $2.35, an increase of 23% over the third quarter of 2012.

28 Oct 2013

State highlights: 'Habilitation' an essential benefit, but what is it?; Va. gov. candidates differ on mental health system; Calif. pays $2.7M for late hearings

A selection of health policy stories from Virginia, California, Colorado, Missouri, Pennsylvania, Kansas and Texas.

25 Oct 2013

DSM, Sanquin Make Factor IX-Recovering Mab Manufacturing Deal

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24 Oct 2013

New smartphone app for patients with hemophilia and related bleeding disorders

BioRx, a national specialty pharmacy and infusion services provider, announced the launch of its industry's first smartphone app designed for patients with hemophilia and related bleeding disorders.

23 Oct 2013

TSRI scientists identify set of compounds to treat multiple sclerosis in new way

Scientists at The Scripps Research Institute (TSRI) have identified a set of compounds that may be used to treat multiple sclerosis (MS) in a new way. Unlike existing MS therapies that suppress the immune system, the compounds boost a population of progenitor cells that can in turn repair MS-damaged nerve fibers.

10 Oct 2013

Catalyst, Isu Abxis To Develop Hemophilia B Candidate

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9 Oct 2013

Alnylam advances Development Candidate for ALN-AS1 for treatment of hepatic porphyrias

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that it is advancing its Development Candidate for ALN-AS1, an RNAi therapeutic targeting aminolevulinate synthase-1 (ALAS-1) for the treatment of hepatic porphyrias including acute intermittent porphyria (AIP).

9 Oct 2013

Roadmap of protein interactions may lead to new therapeutic approaches for type 2 diabetes

A team led by scientists at The Scripps Research Institute (TSRI) has created the first comprehensive roadmap of the protein interactions that enable cells in the pancreas to produce, store and secrete the hormone insulin.

1 Oct 2013

FDA expands Hizentra administration options for people with primary immunodeficiency

CSL Behring announced today that the U.S. Food and Drug Administration has expanded the administration options for Hizentra, Immune Globulin Subcutaneous (Human), 20% Liquid, to include dosing once every two weeks (biweekly) for people diagnosed with primary immunodeficiency.

28 Sep 2013

Japanese MHLW approves Hizentra for treatment of primary and secondary immunodeficiencies

CSL Behring announced today that the Japanese Ministry of Health, Labour and Welfare (MHLW) has approved Hizentra (Immune Globulin Subcutaneous [Human]) for the treatment of primary immunodeficiency (PID) and for the treatment of secondary immunodeficiency (SID). Hizentra is now the first and only 20 percent subcutaneous immunoglobulin (SCIg) therapy in the world for the treatment of these conditions, both of which belong to group of rare and serious diseases of the immune system.

27 Sep 2013

Hemophilia News and Research

Further Reading

Study brings effective hepatitis C cure closer to reality

Human Treg responses allow sustained recombinant adeno-associated virus-mediated transgene expression

Generic drug gabapentin appears to be safe, effective in treating alcohol dependence

Researchers overcome obstacle to using viruses to deliver therapeutic genes

Biogen Idec Q3 total revenues increase 32% to $1.8 billion

State highlights: 'Habilitation' an essential benefit, but what is it?; Va. gov. candidates differ on mental health system; Calif. pays $2.7M for late hearings

DSM, Sanquin Make Factor IX-Recovering Mab Manufacturing Deal

New smartphone app for patients with hemophilia and related bleeding disorders

TSRI scientists identify set of compounds to treat multiple sclerosis in new way

Catalyst, Isu Abxis To Develop Hemophilia B Candidate

Alnylam advances Development Candidate for ALN-AS1 for treatment of hepatic porphyrias

Roadmap of protein interactions may lead to new therapeutic approaches for type 2 diabetes

FDA expands Hizentra administration options for people with primary immunodeficiency

Japanese MHLW approves Hizentra for treatment of primary and secondary immunodeficiencies

Combination of Baxter and Gambro enable Baxter to better serve innovative renal products and therapies

TSRI scientists discover isoform of angiomotin protein responsible for tumorigenesis

UTHealth researchers find cause for rare type of bleeding disorder

Sangamo BioSciences signs definitive agreement to buy Ceregene

FDA grants Orphan Drug Designation to Alnylam ALN-AT3 for treatment of hemophilia B

TSRI scientists discover molecular mechanism by which deadly Ebola virus assembles

Designing fluidic architecture for scalable diagnostic platforms

Evaluating new approaches for rare disease genomics

Expanding access to NMR with the Fourier 80 Duo

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