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.

Cow antibodies points to new ways for making human medicines

Humans have been raising cows for their meat, hides and milk for millennia. Now it appears that the cow immune system also has something to offer. A new study led by scientists from The Scripps Research Institute (TSRI) focusing on an extraordinary family of cow antibodies points to new ways to make human medicines.

7 Jun 2013

Duke researchers find novel way to repair gene responsible for Duchenne muscular dystrophy

Using a novel genetic 'editing' technique, Duke University biomedical engineers have been able to repair a defect responsible for one of the most common inherited disorders, Duchenne muscular dystrophy, in cell samples from Duchenne patients.

5 Jun 2013

PROLOR Biotech announces initiation of hGH-CTP Phase III trial in growth hormone deficient adults

PROLOR Biotech, Inc., a company developing next-generation biobetter therapeutic proteins, today announced the initiation of a pivotal Phase III clinical trial of hGH-CTP, the company's proprietary version of human growth hormone, in growth hormone deficient adults.

4 Jun 2013

New technique selectively represses unwanted immune reactions without disabling immune system

The human immune system is remarkably efficient, but sometimes its attack is misdirected, leading to allergies, autoimmune diseases and rejection of transplant organs and therapeutic drugs. Current immune suppressants have major drawbacks, but a team from The Scripps Research Institute (TSRI) has demonstrated a new technique that may lead to a better way to selectively repress unwanted immune reactions without disabling the immune system as a whole.

4 Jun 2013

Lindsley F. Kimball Research Institute receives grant for global health and development research project

The Lindsley F. Kimball Research Institute of the New York Blood Center announced today that it is a Grand Challenges Explorations winner, an initiative funded by the Bill & Melinda Gates Foundation.

22 May 2013

Biogen Idec seeks FDA BLA approval for PLEGRIDY to treat multiple sclerosis

Today Biogen Idec announced it has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for approval of PLEGRIDY (peginterferon beta-1a), the company's pegylated subcutaneous injectable candidate for relapsing forms of multiple sclerosis (RMS).

21 May 2013

Alnylam reports pre-clinical data from ALN-AS1 program for treatment of AIP

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has presented key pre-clinical proof-of-concept data from its RNAi therapeutic program targeting aminolevulinate synthase-1 (ALAS-1) for the treatment of porphyria including acute intermittent porphyria.

18 May 2013

UIC's Center for Clinical and Translational Science selects six research projects for new pilot grants

The University of Illinois at Chicago's Center for Clinical and Translational Science has selected six research projects to receive pilot grants in 2013.

15 May 2013

Biogen Idec announces FDA's acceptance of ELOCTATE BLA for treatment of hemophilia A

Biogen Idec announced today that the U.S. Food and Drug Administration has accepted the company's Biologics License Application for the marketing approval of ELOCTATE (recombinant factor VIII Fc fusion protein) for the treatment of hemophilia A.

13 May 2013

Scientists identify new way to boost gene-silencing mechanism

A team led by scientists at The Scripps Research Institute (TSRI) has found how to boost or inhibit a gene-silencing mechanism that normally serves as a major controller of cells' activities. The discovery could lead to a powerful new class of drugs against viral infections, cancers and other diseases.

10 May 2013

Immunoglobulin fails to meet co-primary endpoints in Phase III clinical study, Baxter reports

Baxter International Inc. today announced that its Phase III clinical study of immunoglobulin did not meet its co-primary endpoints of reducing cognitive decline and preserving functional abilities in patients with mild to moderate Alzheimer's disease.

From Baxter International Inc. 8 May 2013

Breast milk might prevent premature babies from deadly intestinal condition

An ingredient that naturally occurs in breast milk might be used to prevent premature babies from developing a deadly intestinal condition that currently is largely incurable, according to researchers at the University of Pittsburgh School of Medicine and Children's Hospital of Pittsburgh of UPMC in this week's online early edition of the Proceedings of the National Academy of Sciences.

7 May 2013

TSRI announces five-year agreement with Janssen to research on influenza virus

The Scripps Research Institute (TSRI) today announced a five-year agreement with Janssen Pharmaceuticals, Inc. (Janssen) to collaborate on focused research projects in the infectious disease area, with the initial project targeting the influenza virus.

7 May 2013

TSRI scientists find dissimilar genes that keep very similar shapes

Solving the structure of a critical human molecule involved in cancer, scientists at The Scripps Research Institute have found what they call a good example of structural conservation-dissimilar genes that keep very similar shapes.

2 May 2013

Phase I/II data on Baxter's Lyme disease candidate vaccine presented at NFID conference

Baxter International Inc. today announced Phase I/II data evaluating the safety and immunogenicity of its investigational Lyme disease candidate vaccine during the National Foundation for Infectious Diseases 16th Annual Conference on Vaccine Research in Baltimore, Md.

From Baxter International Inc. 25 Apr 2013

Phase IIb Aviator study demonstrates high SVR rates against genotype 1 HCV

Results from "Aviator," AbbVie's phase IIb clinical trial of its investigational direct-acting antivirals for the treatment of hepatitis C virus infection, continue to demonstrate high sustained viral response rates against genotype 1 HCV, across patient types.

24 Apr 2013

Hemophilia News and Research

Further Reading

Cow antibodies points to new ways for making human medicines

Duke researchers find novel way to repair gene responsible for Duchenne muscular dystrophy

PROLOR Biotech announces initiation of hGH-CTP Phase III trial in growth hormone deficient adults

New technique selectively represses unwanted immune reactions without disabling immune system

Lindsley F. Kimball Research Institute receives grant for global health and development research project

Biogen Idec seeks FDA BLA approval for PLEGRIDY to treat multiple sclerosis

Alnylam reports pre-clinical data from ALN-AS1 program for treatment of AIP

UIC's Center for Clinical and Translational Science selects six research projects for new pilot grants

Biogen Idec announces FDA's acceptance of ELOCTATE BLA for treatment of hemophilia A

Scientists identify new way to boost gene-silencing mechanism

Immunoglobulin fails to meet co-primary endpoints in Phase III clinical study, Baxter reports

Breast milk might prevent premature babies from deadly intestinal condition

TSRI announces five-year agreement with Janssen to research on influenza virus

TSRI scientists find dissimilar genes that keep very similar shapes

Phase I/II data on Baxter's Lyme disease candidate vaccine presented at NFID conference

Phase IIb Aviator study demonstrates high SVR rates against genotype 1 HCV

Scientists reveal that stem cells, tissue-specific cells can be grown from mature mammalian cells

TSRI scientists shed light on major toxic mechanisms of Alzheimer's disease

TSRI scientists identify new drugs for human prion disease

Biogen acquires TYSABRI stake from Elan

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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