Sentences with phrase «in hemophilia»

BioMarin's gene therapy reduces bleeding in hemophilia patients, making Roche look over its shoulder

Adenovirus - associated virus vector - mediated gene transfer in hemophilia B. N Engl J Med 365 (25): 2357 - 65, 2011.

A zymogen - like factor Xa variant corrects the coagulation defect in hemophilia.

Studies investigating the use of emicizumab in hemophilia A patients without inhibitors are underway.

«The first clinical investigation of this drug in hemophilia A patients with or without FVIII inhibitors has already been implemented, and Phase III studies are being planned to start in the near future.»

Formerly a research leader at CHOP, High pursued groundbreaking preclinical investigations in hemophilia B gene therapy and provided scientific expertise to previous gene therapy trials in hemophilia and other genetic disorders at CHOP before moving to Spark Therapeutics, which was spun off from CHOP in 2013.

This discovery may trigger a reassessment in hemophilia research.

For nearly two decades, gene therapy researchers have focused on delivering a gene carrying the code to produce the deficient clotting factor in hemophilia.

In hemophilia, patients are unable to properly control bleeding.

Celgene and bluebird bio report a stunning success in multiple myeloma, Juno Therapeutics takes it on the chin despite positive data, and Spark Therapeutics suffers in its showdown with BioMarin in hemophilia.

Celgene consolidates CAR - T and Sanofi hooks - up in hemophilia.

Not exact matches

Spark Therapeutics» stock soared 20 % in Wednesday trading on the heels of its second quarter 2017 earnings report and some (very) early data on the gene therapy - focused firm's treatment for the blood disorder hemophilia A.

In November, Denner helped facilitate the sale of hemophilia drug maker Bioverativ, for $ 11.6 billion, to Sanofi, a little more than a year after the company was spun out of Biogen, of which Denner is a director.

Last week, Sanofi agreed to buy U.S. hemophilia specialist Bioverativ for $ 11.6 billion, its biggest deal for seven years and a major play to strengthen its presence in treatments for rare diseases.

In the early hours of Monday morning, French drug giant Sanofi said it would buy hemophilia drugmaker Bioverativ, a Biogen spinout that is less than a year old, for $ 11.6 billion.

In January, Sanofi agreed to acquire Biogen's hemophilia spinoff Bioverativ for $ 11.6 billion, and now the Japanese drugmaker Takeda is bidding for the rare - disease drugmaker Shire.

One reason companies like Shire are back to being the hot takeover targets has a lot to do with disease areas they're working in, such as hemophilia, a rare blood disorder.

In recent years, living with hemophilia had become much easier due to advances in treatment and carIn recent years, living with hemophilia had become much easier due to advances in treatment and carin treatment and care.

In a very real sense these brave children with hemophilia or severe allergies or diabetes or cancer, and their brave parents, are reality - checks to the rest of us and our media - inflamed fears of boogy men, eyes getting poked out, and heads being split open.

In a study published earlier this year, his team, along with scientists at Arcturus Therapeutics, treated hemophilia in mice using mRNA that encodes a clotting proteiIn a study published earlier this year, his team, along with scientists at Arcturus Therapeutics, treated hemophilia in mice using mRNA that encodes a clotting proteiin mice using mRNA that encodes a clotting protein.

«In crafting new treatments for hemophilia, a «less is more» approach.»

Patients with hemophilia A have a deficiency of clotting factor VIII (FVIII), while hemophilia B patients are deficient in clotting factor IX (FIX).

Scottish researchers took a similar approach to clone sheep that will produce in their milk human factor IX protein, which is used to treat hemophilia.

Inheriting a single X chromosome exposes men to a host of X-linked diseases, such as hemophilia or Duchenne muscular dystrophy, and researchers hope to use the new data to understand more fully the role of genes in other X-linked conditions.

The advance, described in the current issue of the Proceedings of the National Academy of Sciences, could lead to more powerful and cheaper treatments for hemophilia.

Moreover, the results may transcend the bloating and discomfort of lactose intolerance: The technique could serve as a way to transform the gut lining into a factory for replacing missing proteins in other genetic disorders such as hemophilia, says pulmonologist Eric Alton of the Imperial College School of Medicine in London.

In the lab, replacing malfunctioning genes with working ones has succeeded in curing or treating animals with hemophilia, (the animal analogs of) depression, and other afflictionIn the lab, replacing malfunctioning genes with working ones has succeeded in curing or treating animals with hemophilia, (the animal analogs of) depression, and other afflictionin curing or treating animals with hemophilia, (the animal analogs of) depression, and other afflictions.

Imagine if genetic diseases could be removed from the very biological code of our species — a future in which the likes of hemophilia, cystic fibrosis or dozens of other afflictions are simply edited out of human embryos.

Yet Bennett and her team had been anxious as their study unfolded because AAV (like the early versions of engineered AdV) had previously failed to cure hemophilia in a trial conducted by their Children's Hospital colleague, hematologist Kathy High.

High's gene therapy had cured hemophilia in mice and dogs and had caused rhesus monkeys to express clotting factors.

Early hope for unprecedented cures gave way to tragic deaths, unexpected cancers, and painfully disappointing results in treating hemophilia and HIV.

For instance, from the manufacturer's point of view, an «improvement in the safety of health care» and a «better integration of patients with hemophilia into a normal social life» already arise through market entry.

Turoctocog alfa (trade name: NovoEight) has been approved since November 2013 for the prevention and treatment of bleeding in patients with hemophilia A.

«Turoctocog alfa in patients with hemophilia A: Added benefit not proven, article finds.»

The tumors in Sand's lab might be explained by the high dose, the fact that the mice were newborns, and the type of mouse, suggests Kay, who was involved with a past clinical trial administering AAV to the liver to treat hemophilia.

The method, reported in the November issue of Nature Biotechnology, could lead to safe and effective human gene therapies for cystic fibrosis, hemophilia, and a variety of other diseases.

This is why hemophilia A is almost exclusively seen in men, as they only have one X chromosome in their DNA.

It was hemophilia B, which affects about 1 in 25,000 males in the U.S. — that's five times fewer sufferers than hemophilia A.

In the coming years, we're working hard to bring new therapies to market for patients with MS, amyotrophic lateral sclerosis (ALS, or Lou Gehrig's Disease), hemophilia, and others.

In 2017, a steady stream of encouraging clinical results showed progress in gene therapies for hemophilia, sickle - cell disease, blindness, several serioIn 2017, a steady stream of encouraging clinical results showed progress in gene therapies for hemophilia, sickle - cell disease, blindness, several serioin gene therapies for hemophilia, sickle - cell disease, blindness, several serious

In current treatment, patients with hemophilia give themselves intravenous doses of factor IX up to a couple times a week.

Researchers are reporting the highest and most sustained levels to date of an essential blood - clotting factor IX in patients with the inherited bleeding disorder hemophilia B.

Sufferers of hemophilia live in a perpetual state of stress and anxiety: their joints wear down prematurely and they have bleeding episodes that feel like they will never end.

Thanks to a breakthrough led by researchers in the Cockrell School of Engineering at The University of Texas at Austin, treatment for hemophilia can now be administered via a biodegradable system, a capsule, giving people affected by the hereditary bleeding disorder hope for a less expensive, less painful treatment option than conventional injections or infusions.

Young and his colleagues found that twice - weekly infusions of this novel therapy were well tolerated and resulted in a low incidence of bleeding events in children with severe hemophilia A.

Because their blood does not clot properly, people with hemophilia A have a high risk of excessive internal and external bleeding and may suffer complications in the joints, muscles, and organs.

An antibody engineered to prevent excessive bleeding in patients with severe hemophilia A may be safe and effective, and require fewer injections than existing options, according to a first - in - human study of the treatment published online today in Blood, the Journal of the American Society of Hematology (ASH).

«While an oral delivery platform will be beneficial to all hemophilia B patients, patients in developing countries will benefit the most,» said Sarena Horava, the study's lead author who is a recent Ph.D. graduate from the Cockrell School's McKetta Department of Chemical Engineering and a National Science Foundation graduate research fellow.

«Antibody for severe hemophilia a may reduce injections needed to prevent bleeding: First - in - human study of ACE910 suggests excessive bleeding prevention is possible with a weekly subcutaneous injection compared to standard two or three intravenous injections every week, and should be effective in treating patients with inhibitors.»

In this trial, we demonstrated the safety and efficacy of a medication that will allow children with severe hemophilia A to be treated with a safe and effective medicine but with a reduced burden of the treatment.»