Sentences with word «haemophilia»

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.

Although the dish contains pellets and wet food, there is also a sprinkling of green powder — the product of a trailblazing experiment to address a potentially lethal complication of haemophilia treatment.

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

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

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.

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.

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.

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.

«The challenges of treating haemophilia with inhibitors are just staggering,» says Timothy Nichols, director of the Francis Owen Blood Research Laboratory at the University of North Carolina at Chapel Hill, which maintains the colony of haemophiliac dogs to which Anita belongs.

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.

The technique has also shown promise in treating other genetic diseases such as haemophilia A, cystic fibrosis and some cancers.

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.

The main attraction for Novo is Ablynx's experimental drug caplacizumab for the rare bleeding disorder acquired thrombotic thrombocytopenic purpura, which would complement Novo's line - up of blood products focused on haemophilia.

The French pharma giant is buying U.S. haemophilia group Bioverativ at a whopping 64 pct premium.

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.

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

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.

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.

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.

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.

«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.

People with the inherited bleeding disorder haemophilia lack factors that cause the blood to clot.

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

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

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.

«It's a big improvement,» says Timothy Nichols, a cardiologist and pathologist who studies haemophilia at the University of North Carolina at Chapel Hill.

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

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

The $ 11 billion haemophilia market is facing upheaval and Novo stands to lose sales following approval of a new Roche drug Hemlibra.

A deal would significantly boost Takeda's position in gastrointestinal disorders, neuroscience, and rare diseases, including a blockbuster haemophilia franchise.

Existing haemophilia treatments tip the balance towards clotting by adding what the body lacks — the clotting factor that is missing or defective.

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

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 common thinking among haemophilia treaters is that these new strategies can never replace treatment with factor VIII and IX in non-inhibitor patients,» says Erik Berntorp, a haematologist at Lund University in Malmö, Sweden.

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

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.»

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.

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.

The bioreactor could also provide the means to cure hereditary disorders such as sickle cell anaemia and haemophilia through gene therapy.

«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 hope was that by inserting this gene into patients» cells, it would produce sufficient Factor IX to correct for haemophilia even when it was only infused at low levels.

CHOP present clinical trial at ASH in which gene editing helps haemophilia patients safely maintain clotting factor expression that stops bleeding

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].

Dogs, and other animals can suffer from haemophilia too.

Reasonable excuse can be a physical inability to produce a specimen, eg bronchitis or asthma if requested to produce a specimen of breath, haemophilia if blood is required.