The haemophilia treatment drug Hemlibra is set to take up a dominant position on the market as it dramatically reduced bleeding in test subjects on Monday. The drug, develop by Switzerland-based pharmaceutical company Roche, cut the incidence of treated bleeds in haemophilia A patients who did not get preventive treatment by 96 per cent, compared to 68 per cent in patients who did get preventive treatment. Read more: Alliance Pharma extends Venture Life contract Sandra Horning, chief medical officer at Roche, said: “These new pivotal show that Hemlibra controlled bleeds in people with haemophilia A, while offering the flexibility of less frequent subcutaneous dosing options.” “With this data, we now have positive results from all four of our phase III trials that reinforce the overall efficacy and safety of Hemlibra and its potential to improve care for all people with haemophilia A.” Hemlibra was first successful and approved in patients with inhibitors, … [Read more...] about Roche reveals promising Hemlibra results, predicted to become dominant haemophilia drug
World haemophilia day
A stunning mural has been unveiled in Dublin to mark World Haemophilia Week and the 50th anniversary of the Irish Haemophilia Society (IHS).Contemporary artist Shane O'Malley has created the patient-inspired mural to help to raise awareness of the blood disorder and the incredible work carried out by IHS.O'Malley spoke with both patients and nurses at St James's Hospital, Dublin and used the conversations to help inform his interpretation of haemophilia.“One thing that struck me was the community aspect and how they work together to get the best treatment for patients," said O'Malley."That was really a standing point for me for the mural and I was inspired by that. "This has been a great project to work on, and with everyone working together I think the future is really bright for haemophilia in Ireland.”The IHS works to ensure that people affected by the lifelong blood disorder have access to treatment and support to provide them with more normality in their day-to-day … [Read more...] about Stunning mural highlighting haemophilia unveiled in Dublin
PUBLISHED: 07:00 19 December 2017 | UPDATED: 11:41 19 December 2017 Mike Brooke Haemophiliac Jake Omer after therapy treatment at home with his young family. Picture: Barts NHS Trust Barts NHS Trust A father-of-two diagnosed with haemophilia when he was two-years-old has been given the best Christmas present ever by medical researchers in Whitechapel who appear to have found a breakthrough cure. End in sight to constant injections for haemophiliacs after Barts Health NHS Trust clinical trials. Picture: Nathan Clarke Haemophilia is one step closer to being cured following results of a groundbreaking gene therapy trial at the Royal London Hospital in Whitechapel. One of the test patients was Jake Omer, 29, who has had to have injections three times a week nearly all his life to prevent unstoppable bleeding. His condition had left him with arthritis before he was treated with the gene therapy at the Royal London. “The therapy has changed my life,” Jake said. … [Read more...] about Haemophilia breakthrough cure by Royal London Hospital to missing blood-clotting protein
Home / News / UK and world by Press Association January 1, 2018, 11:14 am FacebookTwitterLinkedInWhatsAppEmail Sign up to our Daily newsletter When it comes to scientific achievements, 2017 certainly left its mark. From the use of ground-breaking gene-editing techniques that can potentially stop inherited diseases from taking root, to the creation of an artificial womb, here are a few of last year’s most important scientific milestones that could potentially change our future. 1. Editing genes to treat inherited diseases Gene-editing technology has been in existence for a few years now, but in 2017 scientists achieved a significant breakthrough when they fixed a mutation responsible for congenital heart disease. Taking advantage of the revolutionary tool called CRISPR-Cas9, a gene linked to hypertrophic cardiomyopathy (a type of heart failure) was successfully “deleted” from a human embryo so they were no longer programmed to inherit the disease. … [Read more...] about 12 of the biggest scientific breakthroughs of 2017 that might just change the world
BREAKTHROUGHS in gene editing have paved the way for scientists to fix "faulty" DNA in humans, potentially wiping out inherited diseases for good.Here is all you need to know about the revolutionary CRISPR-Cas9 technique, what it means for future treatments - and fears it could lead to designer babies.For decades scientists have been looking at ways to alter DNA - the blueprint for all living things.This complex string-like structure is made up of thousands of molecules called nucleotides. Sections of DNA are called genes.The order of the nucleotides creates a kind of biological code that regulates chemical processes, development and growth and determines the physical characteristics of the organism.Genes are passed down from parents to their offspring, and any mutations or mistakes in the DNA sequence can lead to deformities and genetic illnesses affecting the vital organs.Scientists have successfully found ways to manipulate DNA at a molecular level - such as genetically modifying … [Read more...] about What is CRISPR-Cas9, how does gene editing work, who discovered the technique and could it mean we live longer?