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.

New study shows how SNO reaction may contribute to Parkinson's disease

A new study from The Scripps Research Institute is the first to show precisely how a process in nerve cells called the S-nitrosylation (SNO) reaction- which can be caused by aging, pesticides and pollution- may contribute to Parkinson's disease.

22 Nov 2017

FDA approves new drug to treat bleeding in hemophilia A patients

The U.S. Food and Drug Administration today approved Hemlibra (emicizumab-kxwh) to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients with hemophilia A who have developed antibodies called Factor VIII (FVIII) inhibitors.

17 Nov 2017

New pain relievers reduce opiate overdose risk

Scientists on the Florida campus of The Scripps Research Institute (TSRI) have developed new opioid pain relievers that reduce pain on par with morphine but do not slow or stop breathing-;the cause of opiate overdose.

16 Nov 2017

FDA permits marketing of first electric stimulation device to reduce opioid withdrawal symptoms

Today, the U.S. Food and Drug Administration granted a new indication to an electric stimulation device for use in helping to reduce the symptoms of opioid withdrawal.

16 Nov 2017

New drug shows promise in mouse model of autism disorder

Scientists have performed a successful test of a possible new drug in a mouse model of an autism disorder. The candidate drug, called NitroSynapsin, largely corrected electrical, behavioral and brain abnormalities in the mice.

14 Nov 2017

Ozanimod effective against multiple sclerosis, enters New Drug Approval process with FDA

Celgene Corporation recently announced results from two phase 3 trials evaluating the efficacy and safety of the drug ozanimod.

10 Nov 2017

New category of immunotherapy appears safe to use in patients with both cancer and HIV

A new category of immunotherapies called checkpoint inhibitors that has been highly effective against many different cancers appears safe to use in patients with both advanced malignancies and HIV, a population excluded from earlier trials of such therapies, according to an early-phase trial.

7 Nov 2017

Study opens door to new approach for curing HIV infections

In findings that open the door to a completely different approach to curing HIV infections, scientists from the Florida campus of The Scripps Research Institute have for the first time shown that a novel compound effectively suppresses production of the virus in chronically infected cells, and prevents viral rebound, even when those infected cells are subjected to vigorous stimulation.

17 Oct 2017

FDA advisory committee vote 16-0 for first even gene therapy for rare blindness

A panel of advisors to the United States Food and Drug Administration (FDA) has voted unanimously in favor of an experimental gene therapy to treat patients with a rare kind of hereditary blindness.

16 Oct 2017

TSRI study offers key insights into possible links between sleep and hunger

A new study from scientists on the Florida campus of The Scripps Research Institute offers important insights into possible links between sleep and hunger-;and the benefits of studying the two in tandem.

13 Oct 2017

TSRI scientists demonstrate repurposing of DNA to create new substances

DNA evolved to store genetic information, but in principle this special, chain-like molecule can also be adapted to make new materials. Chemists at The Scripps Research Institute have now published an important demonstration of this repurposing of DNA to create new substances with possible medical applications.

12 Oct 2017

New microcapsules represent promising vectors for application of genome-editing tools

Researchers from Tomsk Polytechnic University jointly with their colleagues from St. Petersburg, Hamburg and London have conducted a study in the course of which it was found out that polymer and hybrid silica-coated microcapsules are more efficient in genome-editing when applying CRISPR-Cas9 system.

6 Oct 2017

TSRI scientists find new approach to reprogram ordinary adult cells into IPSCs

Scientists at The Scripps Research Institute have found a new approach to the "reprogramming" of ordinary adult cells into stem cells.

12 Sep 2017

Pfizer announces launch two new digital innovations to help people living with hemophilia

Pfizer Inc. today announced the launch of Hemocraft, a modification of the popular video game Minecraft, and the HemMobile Striiv Wearable, a custom wristband, to help people living with hemophilia better approach hemophilia education and activity tracking in the US.

26 Aug 2017

Shire partners with MicroHealth to support secure care monitoring tool for hemophilia patients with inhibitors

Shire plc, the biotech leader in rare diseases, today announced a collaboration with MicroHealth to support a free and secure care monitoring tool for hemophilia A and B patients with inhibitors and their care teams.

26 Aug 2017

Researchers visually capture 'shapeshifter' that regulates blood clotting

We are normally born with a highly sophisticated array of molecules that act as "sentries," constantly scanning our bodies for injuries such as cuts and bruises.

23 Aug 2017

Researchers investigate genetic mutations in sperm cells to help prevent birth defects

It's about preventing birth defects. Associate pharmaceutical sciences professor Wenfeng An and his team at South Dakota State University are investigating mobile DNA segments, known as L1s, in sperm cells with the goal to treat families at risk for genetic mutations before they conceive.

10 Aug 2017

FDA approves new combo drug Mavyret for adults with Hepatitis C infection

Yesterday the U.S. Food and Drug Administration gave its approval to Mavyret (glecaprevir and pibrentasvir) for the treatment of adults with chronic hepatitis C infection. These patients are infected with the Hepatitis C virus (HCV) of genotypes 1-6 and are those that have not developed liver cirrhosis or those who have developed mild cirrhosis. The approval for Mavyret was given to AbbVie Inc.

3 Aug 2017

Gene-therapy administered through skin transplants could enable treatment of many diseases

A research team based at the University of Chicago has overcome challenges that have limited gene therapy and demonstrated how their novel approach with skin transplantation could enable a wide range of gene-based therapies to treat many human diseases.