Kate Cardente holds her then 3-month-old daughter, Ainsley, as she undergoes a gene therapy for Spinal Muscular Atrophy (SMA). SMA is a disease that is the primary genetic cause of death in babies. […] Life-changer Stevens said the deal was a “life-changer for youngsters with this cruel disease and for their families.” […] The deal struck with Novartis Gene Therapies secures the drug for NHS patients in England at a substantial confidential discount and paves the way for the National Institute for Health and Care Excellence (NICE) — the public body which issues guidance on the cost-effectiveness of drugs and treatments used by the NHS — to publish draft guidance recommending treatment with Zolgensma. […] This approach is backed by NICE given the importance of administering the one-off treatment as early as possible, it added.
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