Month: February 2019

February 27, 2019
Roche invests in gene therapy

Spark Therapeutics, the company at the forefront of developing gene therapy for haemophilia, has been acquired by Roche in a merger worth $4.8 billion. The deal, which is expected to be closed by the middle of the year, brings benefits to both sides. Spark gains the global resources required to fully exploit its discoveries. Roche …

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February 20, 2019
EHC announces first conference on women and rare bleeding disorders

The European Health Consortium (EHC) has announced that its first European Women & Bleeding Disorders Conference will be held in Frankfurt, Germany, on May 24th– 26th. The programme will cover issues in genetics, management of heavy menstrual bleeding, family planning, everyday coping, anaemia, oral care, impact on quality of life and pregnancy. Online registration is …

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February 19, 2019
Virtual reality distracts children during venepuncture

US researchers have developed a hands-free virtual reality environment that could help to avoid the development of needle phobia in children with haemophilia (JMIR Serious Games. 2019 Jan 9;7(1):e10902. doi: 10.2196/10902). It was no more effective than a standard technique but probably more fun. They created a wireless virtual reality platform using a disposable adjustable …

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Is fortnightly FIX feasible?

Bioverativ’s B-LONG and B-YOND studies demonstrated the long-term safety and efficacy of its EHL FIX, rFIXFc, when administered every 7 days to >14 days. Now, a post hoc analysis of the small subgroup of patients who used a dose interval of 14 days or longer has shown that control of bleeding was similar to that …

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February 18, 2019
EHL FIX review

An up to date review of evidence for the efficacy and safety of nonacog beta pegol, an extended half-life FIX for haemophilia B, covers outcomes from the PARADIGM trial programme (Drug Des Devel Ther 2018;12:2933-2943). The review is free to access at

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February 17, 2019
Is EHL factor replacement therapy cost effective?

Health economists continue to grapple with the issue of cost effectiveness of extended half-life (EHL) factors compared with the established recombinant factors. A report from the United States suggests that, for people with haemophilia A, they are not (Manag Care 2018;27:39-50). Analysis of data over a two-year period before and after a switch to an …

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February 14, 2019
Obesity and haemophilia

It’s becoming increasingly clear that, despite some obvious differences directly attributable to their bleeding disorder, people with haemophilia share many of the problems that plague the population in general. A survey of seven haemophilia centres in Germany has shown that the prevalence of overweight and obesity is similar to that in the population as a …

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February 9, 2019
Phase III gene therapy trial begins and Phase II trial updated

The first patient in uniQure’s HOPE B trial of its gene therapy AMT-061 has been treated, the company has announced ( HOPE B is an non-blinded Phase III trail to determine the efficacy and safety of AMT-061 in men with haemophilia B; it will eventually involve a total of 56 patients.The trial comprises a six-month …

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February 7, 2019
Roche joins WFH Humanitarian Aid Program

Roche has joined the World Federation of Hemophilia’s Humanitarian Aid Program with a donation of emicizumab and funds to support training to build expertise in economically developing countries ( The donation will provide prophylaxis with emicizumab for up to 1,000 people in over five years, focusing on high-need patients such as adults with inhibitors and …

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February 5, 2019
Calling all high-flying researchers

Funding of up to £2 million is being made available to researchers ‘with compelling track records in the health sector’ with ‘potentially high-risk projects… grounded in considerable external evidence’. The Peter Sowerby Foundation is inviting applications from organisations with a turnover of more than £2 million with projects ‘such as primary medical care and research …

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