Sentences with phrase «haemophilia at»

Not exact matches

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.

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.

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.

«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.