Sentences with phrase «in haemophilia»

Indeed, psychological interventions have been proven to be effective in a broad range of disorders and illnesses.15 — 18 Although a few former works have focused on psychological interventions in haemophilia, showing positive and promising results, 19 — 25 the lack of recent papers exploring this issue is somewhat surprising, despite the recommendations and guidelines that emphasise their relevance.

The current study protocol points to an innovative research that can contribute to better understand the impact and potential benefits of psychological interventions in haemophilia care setting.

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.

The hunt is on for ways to diagnose and treat the joint problems that are now the main chronic problem in haemophilia

Depending on the person, the amount of factor VIII — the protein missing in haemophilia A — in the bloodstream drops by half in a mere 8 — 12 hours.

Not exact matches

Last week, Sanofi agreed to buy U.S. haemophilia 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.

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

In the same year that Novo Nordisk launched its concizumab trial, Baxter struck a deal to purchase a suite of haemophilia - related assets from the former therapeutics company Archemix.

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.

Drug company Baxter International, based in Deerfield, Illinois, sells recombinant clotting factors for treating haemophilia and also has its sights on TFPI.

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

«These are remarkable data,» says Amit Nathwani at University College London, who is using AAVs in potential treatments for a blood disorder called haemophilia B. «Liver fibrosis is a major clinical problem and if these data can be reproduced clinically, the National Health Service would save billions and patients would be given a new lease of life.»

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.

Experiments in mice suggest that treatment of haemophilia could be more successful if the baby's immune system is primed while in the womb

Indeed, exposure of the protein produced by the nanoparticle - based gene therapy to the gut mucosa prevents inhibitor development and restores clotting - factor activity in mouse models of both haemophilia A and B. «This approach really could hold big benefit for patients,» says Jörg Schüttrumpf, a transfusion - medicine specialist who led one of the studies performed at the German Red Cross Blood Donor Service in Frankfurt.

He focused first on haemophilia B. Adapting a technique that he had previously developed to delay the onset of type 1 diabetes, Daniell and his group genetically modified tobacco plants to express human factor IX in their chloroplasts.

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.

Earlier this year, the two researchers and their teams documented suppression of inhibitor formation and even reversal of pre-existing inhibitors in mouse models of haemophilia A.

This led to long - lived tolerance in mouse models of haemophilia A and B.

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.

Shire CEO Flemming Ornskov - who has a large conventional haemophilia business and is also chasing Biomarin and Spark in hunting a cure for the bleeding disorder - sees both the opportunities and the difficulties of gene therapy.

«In an area like haemophilia I think that approach is going to make a ton of sense, since the budget impact there starts to get more significant,» Marrazzo said.

After decades of frustrations, firms believe there are now major opportunities for gene therapy in treating inherited conditions such as haemophilia.

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

In Madrid last week, Amit Nathwani of the Royal Free NHS Trust in London announced that six people treated for haemophilia using AAV in early 2011 are still producing the blood clotting factor they previously lackeIn Madrid last week, Amit Nathwani of the Royal Free NHS Trust in London announced that six people treated for haemophilia using AAV in early 2011 are still producing the blood clotting factor they previously lackein London announced that six people treated for haemophilia using AAV in early 2011 are still producing the blood clotting factor they previously lackein early 2011 are still producing the blood clotting factor they previously lacked.

Disorders like haemophilia that are caused by faults on the X chromosome are therefore more common in males.

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.

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.

A «cure» for haemophilia is one step closer, following results published in the New England Journal of Medcine of a groundbreaking gene therapy trial led by the NHS in London.

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

Professor Pasi continued: «Incredibly exciting is the potential for a significant change in how haemophilia is treated globally.

Almost exactly a year ago, we reported on a gene therapy for haemophilia that was in development by the Children's Hospital of Philadelphia (and subsequently the CHOP spinout Spark Therapeutics).

The results, published in the New England Journal of Medicine, demonstrate the therapy being used to treat 10 male haemophilia patients without significant side effects.

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

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

Similar to haemophilia in humans, von Willebrand's disease can result in life - threatening bleeding.

They can also suffer from a disease called Von Willebrand's Disease, which is similar to haemophilia in humans.

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).

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.