Medicine regulators yesterday approved gene therapy for the condition Spinal muscular atrophy and it will soon be available on the NHS.
A miracle ‘cure’ for a savage disease that, in its most severe form, kills babies before they turn two, will soon be on theSpinal muscular atrophy is a genetic condition that robs people of their ability to sit, crawl, walk, talk, swallow and breathe.
The one-time treatment in the US costs $2.1 million, or around £1,800,000, making it one of the most expensive drugs in the world. But the National Institute for Health and Care Excellence has now rubber-stamped the gene therapy for use at the early presymptomatic stage. His mum Natalie Greenhall, 33, said: ‘From being told Theo had one of the most fatal, most horrible diseases, we now hope to see many birthdays, a lot of holidays, and that he will outlive us as parents.’
The test screens for nine rare health conditions, such as cystic fibrosis, sickle cell disease and severe combined immunodeficiency. But Zolgensma, by Swiss drugmaker Novartis, infuses babies with a harmless virus to help them make a new version of the faulty SMN1 gene.
Belgique Dernières Nouvelles, Belgique Actualités
Similar News:Vous pouvez également lire des articles d'actualité similaires à celui-ci que nous avons collectés auprès d'autres sources d'information.
Martin Lewis shares 'secret' code that could help you save money on medicineMARTIN Lewis has shared a little-known “secret” code that could save you money when buying over-the-counter medicines. The money saving expert, 50, revealed the tip on a recent episode of his Money…
Lire la suite »
NHS Unions 'receive new pay offer'BREAKING: After reports of progress in talks, NHS unions have reportedly received a new pay offer expected to be announced this afternoon.
Lire la suite »
Hopes for end to crippling NHS strikes as ministers offer a 5% pay dealTHE UK government has agreed to a pay deal for nurses and ambulance staff amid a wave of strikes. The revised offer will see the two groups receive a 5% pay rise in 2023-24, according to Bloomberg …
Lire la suite »
NHS unions receive new pay offer - announcement expected this afternoonNHS unions have received a new pay offer and an announcement is expected this afternoon, Sky News understands. Sky's Matthew Thompson has the latest 📺 Sky 501, Virgin 602, Freeview 233 and YouTube
Lire la suite »
NHS strike action by several unions paused after improved pay offerBREAKING: Fresh NHS strikes could be averted after the government put an extra £2.5 billion on the table to fund a pay rise for thousands of healthcare staff. Read more: 📺 Sky 501, Virgin 602, Freeview 233 and YouTube
Lire la suite »
NHS strikes paused after health unions and ministers agree pay offer🔴 Strike action by Unison, GMB, Royal College of Nursing and Unite will be paused while hundreds of thousands of members are balloted over the offer
Lire la suite »