Friday, 24 May 2019

U.S. approves Novartis gene therapy for rare, deadly muscle disorder

NEW YORK/ZURICH - Swiss drugmaker Novartis won U.S. approval for its gene therapy Zolgensma for spinal muscular atrophy (SMA), the leading genetic cause of death in infants, the company said on Friday, a one-time treatment expected to push the pricing boundary for rare diseases to new heights.


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