Sentences with phrase «with haemophilia a»

This is an innovative investigation, applying two empirically sound interventions (cognitive - behavioural therapy and hypnosis) to people with haemophilia.

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).

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

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

«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.

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.

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

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

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.

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

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

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

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

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.

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.

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

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

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

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

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

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

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.

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

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

Not exact matches

Biopharmaceutical treatments, led by haemophilia, make up around 20 percent of Novo's sales, with diabetes and obesity products accounting for the remaining 80 percent.

When he delved into the genomes of those with a milder disease, he often saw not just a mutation in the affected clotting - factor gene, but also a mutation in another gene — the first causing haemophilia, the tendency to bleed, and the second causing thrombophilia, the tendency to clot.

They also found that it could shorten bleeding time and hasten clotting in rabbits with induced haemophilia.

Although its launch in Britain last week could give them access to uncontaminated and potentially limitless supplies, the 95 haemophilia treatment centres may not be able to afford the new drug, which costs 52p per unit, compared with 20 to 45p for factor 8 extracted from human blood.

When injected together with factor VIII into mouse models of haemophilia A, the nanoparticles deliver their payload to cells in the lymphoid tissue that are responsible for initiating immune responses.

2011 Six people with clotting disorder haemophilia B see a reduction in symptoms after gene therapy on liver cells.

One 2001 study suggested that up to 40 % of people with severe haemophilia do not follow the prophylactic schedule.

Eloctate, for haemophilia A, was approved in June and is recommended for an initial infusion once every four days, with a physician adjusting that up to five days or down to three as appropriate.

Replacing the clotting ability lacking in haemophilia has been the treatment since the 1840s, when attempts were made to treat people with the disease by transfusion with whole blood from people with normal clotting.

There are around 2000 people with severe haemophilia A in the UK.

A hereditary genetic condition dominantly affecting men, people with severe haemophilia A have virtually none of the protein factor VIII which is essential for blood to clot.

Some concerns had been raised that by infusing patients with such a high activity gene, the treatment would overcorrect for haemophilia and give rise to thrombosis.

The Kennel Club also recommends testing for haemophilia and hip dysplasia, other common problems with the breed.

Check with your breeder whether their dogs have been screened for haemophilia and hip dysplasia and ask your vet to regularly check your dog's hips.

Another noteworthy issue is that psychological or psychiatric conditions are reported by 47 % of PWH, with 29 % relating these symptoms to haemophilia.4 This is even more relevant considering that psychological factors can influence both pain experience and QoL in PWH.12 Interestingly, Cassis et al 6 state that variations in QoL are better explained by psychosocial, rather than clinical predictors.