Sentences with phrase «with haemophilia»

Only about 4 % of people with haemophilia B develop inhibitors to factor IX.

To investigate these concerns, the study followed 10 male patients with haemophilia B who were all treated with the same low dose of Factor IX - R338L over time.

Factor IX — which people with haemophilia B lack — lasts longer, 18 — 24 hours.

People with haemophilia A, the commonest form of the disease, need infusions of factor 8 because a hereditary gene defect means they can not make their own.

Disclosure of parental HIV infection to children in the families of men with haemophilia: Rates, outcomes, and the role of the family environment.

Ghosh's research leads to the conclusion that a patient with haemophilia who co-inherits a thrombophilic gene bleeds less than one without that mutation.

Parents must infuse a toddler as often as every other day, and children with haemophilia will have to continue that treatment for the rest of their lives.

Kanjaksha Ghosh has seen more than a thousand people with haemophilia since he became a physician.

Sorensen thinks that if they can achieve a 50 — 80 % reduction in antithrombin, ALN - AT3 may be able to control bleeding in people with haemophilia without infusions of clotting factor.

If the strategy also works in Anita and her kennel mates — and ultimately in humans — it could form the basis of the first product to protect against the immune responses associated with haemophilia treatment.

When treated with replacement coagulation proteins, the dog naturally develops antibodies, or inhibitors, against the therapy — a problem that is also seen in some 5 % of humans with haemophilia B.

With so many therapeutic tactics moving through the preclinical pipeline, scientists and clinicians remain hopeful that at least one will ultimately succeed, eliminating the problem of inhibitor formation for people with haemophilia altogether.

Kam Leong, a biomedical engineer at Columbia University in New York City whose team was the first to demonstrate success with this approach in mice, has even tried feeding the chitosan — DNA nanoparticles to dogs with haemophilia A. Leong found some evidence of gene transfer and a reduction in inhibitors in the animals.

Anita, so named because her red coat reminded breeders of the character from the animated film One Hundred and One Dalmatians, is a keagle (a mix of a beagle and a Cairn terrier) with haemophilia B.

Using the standard gene - therapy approach, researchers have shown that they can achieve long - term expression of factor IX in adults with haemophilia B at sufficiently high levels to convert the bleeding disorder into a mild disease (see page S6).

The problem is even worse with haemophilia A, a disease that is four times more common than haemophilia B and in which the missing link in the coagulation chain is a protein called factor VIII.

As these therapies emerge, dealing with haemophilia will become less troublesome (see below).

For the parents of a child born with haemophilia, the diagnosis comes with both good and bad news.

We really now have the potential to transform care for people with haemophilia using a single treatment for people who at the moment must inject themselves as often as every other day.

Clinical researchers at Barts Health NHS Trust and Queen Mary University of London have found that over one year on from a single treatment with a gene therapy drug, participants with haemophilia A (the most common type) are showing normal levels of the previously missing protein, and effectively curing them.

«People who live with haemophilia today face a lifelong need for vigilant monitoring and recurrent factor concentrate infusions to prevent spontaneous, potentially life - threatening bleeds and to protect their joints,» said Katherine High, President and Head of Research and Development at Spark Therapeutics.

Effectiveness of two psychological interventions for pain management, emotional regulation and promotion of quality of life among adult Portuguese men with haemophilia (PSY - HaEMOPEQ): study protocol for a single - centre prospective randomised controlled trial

Spark Therapeutics» gene therapy for patients with haemophilia has seen some early success in clinical trials, after being tested in 10 male patients

For a person with haemophilia A, factor VIII is a foreign substance, and the immune system can see it as a threat.

Studies published over the past two decades suggest that blocking this protein can promote clotting, which could curb bleeding in people with haemophilia.

Baxter, Novo Nordisk and Alnylam think that their products will appeal to other people with haemophilia.

If compounds such as concizumab and ALN - AT3 prove effective, they will undoubtedly be a boon for at least one group of people with haemophilia: those who develop inhibitory antibodies against the blood - clotting factors VIII and IX, and who can no longer receive this standard therapy.

But many of the physicians who treat patients with haemophilia are not convinced.

The next phase of the study will include people with haemophilia, and there will not be the same limitation.

People with haemophilia «are more heterogeneous than we'd like to admit,» he says.

The researchers administered the antibody either intravenously or subcutaneously to 28 healthy volunteers and 24 people with haemophilia.

Roughly 5 % of those with haemophilia B fall into this category, and 30 % of those with haemophilia A (see page S12).

Its researchers showed that this antibody could speed up clot formation in blood plasma from people with haemophilia.

Around 30 % of people with haemophilia A develop antibodies against replacement factor VIII.

Extending the life of clotting factors may improve quality of life for people with haemophilia

The researchers have not yet released the results of their phase II initial clinical trials, but Shima says that in the patients with haemophilia they looked at, bleeding frequency decreased dramatically.

About 30 % of people with haemophilia A develop inhibitors, and once they do, treating their bleeding becomes much more difficult.

Studies dating back to the 1970s have shown that PEGylation can reduce the chances of a foreign protein stimulating an immune reaction, although the effect has not yet been proved in people with haemophilia.

For all the advantages of these extended - life molecules, the researchers predict that they will be supplanted in perhaps a decade by advances in gene therapy, which will enable people with haemophilia to produce their own clotting factors.

Those short half - lives mean that most people with haemophilia must transfuse themselves every two or three days.

«What is truly remarkable about this revolutionary new gene therapy are the profound life - changing effects it offers patients with haemophilia.

Spark Therapeutics» gene therapy for patients with haemophilia has seen some early success in clinical trials, according to a report published yesterday.

Psychosocial health and effective pain management are considered essential end points for optimal haemophilia care, but there is a significant gap in evidence - based treatments targeting these outcomes in people with haemophilia (PWH).