Health
NHS secures world’s most expensive drug to treat babies with rare muscle disease – The Irish News
Patients will be able to access Zolgensma, a one-time gene therapy, said NHS England.
Up to 80 babies a year born with a rare muscle-wasting disease could receive life-changing treatment after the most expensive drug in the world was licensed for use on the NHS in England.
Infants born with Spinal Muscular Atrophy (SMA), an often fatal condition which affects the spinal cord nerves causing paralysis, will be able to access Zolgensma, a one-time gene therapy, said NHS England.
The drug, which replicates a missing gene and restores nerve and muscle function, has a reported list price…
-
Business21 hours agoWhere will Berkshire Hathaway be in 1 year?
-
Noosa News16 hours agoTwo trapped and fighting for life after major three-vehicle crash in Brisbane
-
Noosa News24 hours agoNick Cave and The Bad Seeds Are Bringing Their ‘Wild God’ Tour to Australia and New Zealand in 2026
-
Noosa News23 hours agoBarry Du Bois opens up at the high-tech Hinterland Health Retreat