Long term conditions

NHS England strikes deal on life-saving gene-therapy drug that can help babies with rare genetic disease move and walk

A life-saving drug that can enable mobility in babies and young children suffering from a rare genetic condition will be available on the NHS, chief executive Sir Simon Stevens announced today. Zolgensma, which has a reported list price of £1.79 million per dose and is labelled the most expensive drug in the world, will be […]

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Landmark NHS deal to open up access to life-changing cystic fibrosis drug

From today thousands of NHS cystic fibrosis patients in England can benefit from a ‘transformative’ treatment for cystic fibrosis. NHS patients will be among the first in Europe to be prescribed Kaftrio, which significantly improves lung function, helping people with cystic fibrosis to breathe more easily and enhancing their overall quality of life. The treatment […]

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Life-changing treatment on the NHS for children with rare, deadly disease

Children in England with a rare condition which leads to early death are set to receive treatment on the NHS, after the health service struck a deal with manufacturers. Young people with Batten Disease – an incurable illness which affects the nervous system, causing seizures, visual impairment, mobility loss and early death – will now […]

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England’s top doc backs team GB for transplant games success

England’s top doctor, Professor Stephen Powis, has sent a message of support to Team GB athletes competing in the World Transplant Games this weekend, and joined with them in thanking the donors and skilled NHS teams who made it possible. The NHS’ national medical director, who worked as a kidney transplant specialist, also revealed that […]

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Children’s hospices to receive £25 million a year as part of NHS Long Term Plan

Funding for children’s hospices will rise to £25 million a year, NHS England has pledged. Medical advances mean the NHS can help seriously ill children and young people with more complex health issues live longer, more fulfilling lives. NHS England’s hospice grant programme currently provides £12 million a year for children’s hospices, helping to provide […]

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NHS England to fund first ever treatment for children with rare muscle-wasting condition

NHS England will provide a promising new treatment which can prolong the lives of children with a rare genetic condition after negotiating a deal with the manufacturer. Nusinersen, also called Spinraza and made by Biogen, is the first treatment that targets the underlying cause of spinal muscular atrophy (SMA). The condition affects the nerves in […]

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NHS England strikes deal to make innovative Multiple Sclerosis drug available, paving the way for NICE recommendation

Patients with Multiple Sclerosis (MS) will have routine access to a drug that can slow disability under a deal struck between NHS England and the manufacturer Roche, paving the way for its approval by NICE. Ocrelizumab (also known as Ocrevus) is the first treatment that can modify or change the course of the disease to […]

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