News

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NHS will pay for treatment with emicizumab

NHS England has announced that the NHS will pay for treatment with emicizumab and susoctocog alfa, among several innovative therapies assessed by its Clini...

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Gene therapy begins final trial stage

Pfizer and its biotech partner Spark Therapeutics have announced the beginning of a Phase III trial of their gene therapy for haemophilia B, fidanacogene e...

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Novel treatment for joint pain shows promise

Pfizer has been working with Lilly on the development of a novel treatment for pain, recently announcing positive results in patients with pain associated ...

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Genome editing ‘could be morally permissible’

‘Editing the DNA of a human embryo, sperm, or egg to influence the characteristics of a future person (‘heritable genome editing’) could be morally permiss...

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Mobile haemophilia care

A mobile home visiting service is a valued alternative to attending a centre, say researchers from haemophilia services in Germany (Hamostaseologie 2018 Ju...

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Pharma talks about barriers

Representatives of the pharmaceutical industry are to meet in the US to discuss barriers to the uptake of new therapies for haemophilia. Participants at th...Hemophilia Drug Development conference in Boston in August 2018 will discuss how best to increase patient engagement, the impact of next-generation therapies, factors influencing their uptake, and what endpoints will be important when evaluating innovative drugs. The agenda highlights issues likely to attract research funding in the near future and form the basis of debate with patients, carers and the health professions.

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FVIII/vWF on trial for inhibitors

Wilate, the infusion combining Factor VIII and von Willebrand Factor in equal proportions, is to be evaluated as a treatment for patients with haemophilia ...

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Register now for ECH Inhibitors Summit

The European Haemophilia Consortium has announced the third summit for children and adults with inhibitors to be organised by the Barretstown charity (www....

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High potency Factor IX: inhibitors reported

CB 2679d/ISU304, a high potency recombinant Factor IX that can be given subcutaneously, is undergoing Phase I/II trials that will define the optimal dose a...

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Catching up with reviews

Italian investigators have drawn on literature reviews to describe the impact of developing inhibitors on people with haemophilia A or B (Eur J Haematology...

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UCL fund supports gene technology research

The UCL Technology Fund, established to promote development of innovations arising from University College London’s research programme, has joined with inv...

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Liver transplantation in haemophilia A and von Willebrand disease

Hepatitis C is a major risk factor for liver cirrhosis and hepatocellular carcinoma, which means that people with haemophilia who were infected in the bloo...

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Long-term outcomes after total elbow arthroplasty

Surgeons from Switzerland have reported outcomes after an average of 9 years follow-up of nine patients who received 13 total elbow arthroplasties between ...

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Less cardiovascular disease in men with haemophilia

Men with haemophilia have less cardiovascular disease (CVD) than age-matched controls, say US investigators (Blood Adv 2018;2:1325-1333). They defined ...

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More news from Glasgow

Haemnet was everywhere at the World Federation for Haemophilia Congress in Glasgow, or so it seemed, but there was so much going on that inevitably we miss...

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We’ve never had it so good

Outcomes for people with moderate or severe haemophilia A or B are improving, a Swedish survey has shown (J Rehabil Med 2018 Jun 8. doi: 10.2340/16501977-2...

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A round-up of recent reviews

People with haemophilia are at increased risk of osteoporosis but the underlying mechanism is still uncertain, according to a new review (Blood Rev 2018 Ma...

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Prophylaxis, Canada style

‘Tailored frequency-escalated prophylaxis’ uses less clotting factor than standard protocols and delivers good outcomes, say investigators reporting on a m...

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EAHAD defines principles of inhibitor management

Principles for the management of patients who develop inhibitors have been developed by a multidisciplinary group of health professionals and patients unde...

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Perioperative replacement therapy in haemophilia B

Targeting Factor IX levels in patients with. Haemophilia B who are undergoing surgery is ‘complex and suboptimal’ but the incidence of bleeding complicatio...

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Cost of gene therapies might be millions per patient

The cost of gene therapy for patients with haemophilia could be as high as $2 million per person, according to US analysts. Early estimates of the cost...

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Stem cell therapy for haemophilia B?

US researchers have shown that it is possible to engineer stem cells to correct the gene defect that causes haemophilia B and use them to restore normal cl...

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Stem cell therapy for haemophilia B?

US researchers have shown that it is possible to engineer stem cells to correct the gene defect that causes haemophilia B and use them to restore normal cl...

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Emicizumab in patients without inhibitors

The Food and Drug Administration, the US drug regulatory authority, has granted ‘breakthrough therapy designation’ to Roche’s emicizumab (Hemlibra) to supp...

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Switch to EHL factors costly in US

An analysis by a US health economics firms has found that switching from conventional to extended half-life clotting factors is associated with a doubling ...

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New evidence of the impact of haemophilia on individuals and carers

Two new reports have confirmed how common pain and psychiatric morbidity are among people with haemophilia. B-HERO-S is a US study of 299 adults with h...

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Factor storage affects patient satisfaction

Clinicians should remind patients that clotting factors don’t generally need to be stored in the fridge because, a study from Italy suggests, this may infl...

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Novel biotechnology recognised with special EU status

A technology that could offer people with haemophilia B an implant providing long term Factor IX synthesis has been awarded Advanced Therapy Medicinal Prod...

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Long term outcomes in Finland

In Finland, all children with severe haemophilia are treated according to a single protocol, whether it’s delivered as part of comprehensive care or in a t...

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Factor storage affects patients satisfaction

Clinicians should remind patients that clotting factors don’t generally need to be stored in the fridge because, a study from Italy suggests, this may infl...

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Novel biotechnology gains special EU status

A technology that could offer people with haemophilia B an implant providing long term Factor IX synthesis has been awarded Advanced Therapy Medicinal Prod...

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New evidence of the impact of haemophilia on individuals and carers

Two new reports have confirmed how common pain and psychiatric morbidity are among people with haemophilia. B-HERO-S is a US study of 299 adults with h...

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New human fibrinogen concentrate available

FibCLOT, a new human fibrinogen concentrate is now available in the UK, offering increased therapeutic choices for patients affected by congenital fibrino...

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Emicizumab meets study endpoint

22 December 2016 Prophylaxis with emicizumab reduced the number of bleeds over time compared with no prophylaxis in the HAVEN 1 study, which recruited ...

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Elocta and Alprolix gain NHS reimbursement across the UK

21 September 2016 NHS England has confirmed reimbursement of Elocta® (efmoroctocog alfa) and Alprolix® (efrenonocog alfa) for the treatment of haemophilia ...

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Emicizumab data looks encouraging

29 July 2016 Emicizumab, the humanized bispecific monoclonal antibody, in development by Roche/Genentech, continues to look promising for people with s...

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EMA discounts increased inhibitor risk with Kogenate and Helixate

13 May 2016 Currently available evidence does not confirm that Kogenate Bayer/Helixate NexGen is associated with an increased risk of factor VIII inhib...

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First longer-acting FVIII moves a step closer

28 September 2015 The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has issued a positive recommendation for the ma...Once approved, Elocta will likely be the first prolonged half-life treatment option available for people with haemophilia A in the UK and Ireland.

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EMA validates Alprolix (rFIXFc) marketing authorisation application

26 June 2015 The European Medicines Agency (EMA) has accepted Swedish Orphan Biovitrum AB (Sobi) and Biogen’s Marketing Authorisation Application for Alproli...

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Sobi launches movie-maker app

Children living with haemophilia sometimes have a hard time talking about their condition or describing the implications the disease has on their life. Sob...

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Bayer recruits children to long-acting trial

Bayer HealthCare has started to enroll children in an international phase III trial to evaluate its investigational compound BAY 94-9027 for the treatment ...

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Another one bites the dust

Bad news for Bayer this week. The company has just announced the discontinuation of a Phase II/III trial evaluating the efficacy and safety of BAY 86-6150 ...

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Baxter to Initiate Late-Stage Trial of Longer Acting Advate

Baxter in the US has submitted an Investigational New Drug (IND) application for its investigational haemophilia A treatment BAX 855 with the US Food and D...

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B-LONG results positive for Biogen/SOBI

Wednesday 26 September 2013: Top-line results from the late-stage B-LONG trial suggest that Biogen/SOBIs’ long-lasting recombinant Factor IX Fc fusion prot...

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New kid on the block looks promising

Pro Bono Bio Group plc has successfully concluded its pre-clinical trials in haemophilia patients using the world's first long acting blood Factors VIIa, V...

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EMA Calls for Further Research on Personalized Approaches to Treating Hemophilia

The European Medicines Agency (EMA) has released a new reflection paper on the risks of adverse immune system reactions in hemophilia patients treated with...

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Children to be seen (and heard) at EMA paediatric committee

Scrip reports that the European Medicines Agency is planning to allow children to participate in its paediatrics committee (PDCO) meetings to get their fee...

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Inspiration files new FIX in US

Inspiration Biopharmaceuticals has submitted a Biologics License Application (BLA) to the US Food and Drug Administration (FDA) for the approval of IB1001,...

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First patient screened in CSL Behring global phase I/III study of recombinant single-chain factor VIII

CSL Behring has screened the first patient in its recombinant coagulation single-chain factor VIII (rFVIII) trial, part of the AFFINITY clinical trial prog...

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Inspiration initiates second trial of porcine FVIII

Inspiration Biopharmaceuticals, Inc. has announced the initiation of patient enrolment in the second of two pivotal studies in its OBI-1 Accur8 clinical tr...

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FEIBA prophylaxis tops on-demand treatment for joint bleeds

Prophylaxis with FEIBA (anti-inhibitor coagulant complex, AICC) significantly and safely decreased the frequency of joint and other bleeding events compare...

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Prenatal test extends choice for pregnant women

Pregnant women who carry the haemophilia gene could benefit from a non-invasive prenatal diagnostic test that analyses fetal DNA circulating in the mother’...




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