Sentences with phrase «for haemophilia»

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.

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

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.

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 «transformational» results have particular significance as the first successful gene therapy trial for the haemophilia A.

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.

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.

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.

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.

Protein replacement has been the go - to therapy for haemophilia for decades.

Although the antithrombin - based therapies for haemophilia are not designed to completely block their targets, «knocking them down is not without risk», he says.

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.

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.

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.

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

Men inherit one X and one Y chromosome (which encodes the gene for maleness), so they are more likely to express recessive traits from the X, such as colour blindness and 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.

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.

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.

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.

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.

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

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

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

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.

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.

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.

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

«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.

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.

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 results from the first human trials for a gene therapy to treat haemophilia A have just been published.

For questions regarding breed survey, hip and elbow dysplasia, tattooing, tooth certificates or haemophilia see the Breed Matters Section: Breed Survey, Hip & Elbow Dysplasia, Tattoo, Tooth Certificates, 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).