Sentences with phrase «of haemophilia»

Most of us have heard of haemophilia.

The treatment of haemophilia, dialysis and the medical applications of ultrasound are just a few historical examples.

«We think we can change the whole concept of haemophilia treatment,» Shima says.

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.

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

Pills made from lettuce leaves could help to prevent one of the most serious complications of haemophilia treatment

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.

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.

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

Clotting - factor infusions treat symptoms of haemophilia, but gene therapy could provide a cure.

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.

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.

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

Concizumab and similar therapies represent «a completely different way of approaching haemophilia compared to anything we've been doing for the last 50 years», he says.

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

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.

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

The test developed can be carried out on mothers at risk of X-linked genetic recessive diseases including haemophilia and Duchenne muscular dystrophy and mothers at risk of haemolytic disease of the new - born.

«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.»

Down's syndrome, haemophilia, fragile X syndrome, sickle - cell anaemia, thalassaemia and a plethora of other disorders are also down to genetic defects.

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.

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.

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.

The nanotechnology approach that is being tested for inhibitor control could also improve the haemophilia treatment that is now at the cutting edge of clinical research: gene therapy.

Still, the standard form of liver - targeted gene therapy carries a range of potential complications, including the risk of harmful mutations and of the body mounting an immune response against the viral vectors used to carry the correct forms of the defective genes responsible for haemophilia.

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.

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 lacked.

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

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.

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

One barrier to haemophilia therapy is the tendency of factor VIII to prompt the body into producing anti-factor VIII antibodies, known as inhibitors.

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

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.

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

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.

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.

Ohmori, Tsukasa, et al. «CRISPR / Cas9 - mediated genome editing via postnatal administration of AAV vector cures haemophilia B mice.»

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).

«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.

The therapy hoped to resolve some of the problems surrounding haemophilia treatment by exchanging the continual infusions for a single vector infusion that could permanently boost Factor IX expression.

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.

iPS cells have been employed to generate cells for the treatment of various diseases including diabetes, cardiovascular disease, sickle cell anaemia, Parkinson's disease and haemophilia [19]--[23].

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.

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.

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.