US approves the one-time treatment for deadly spinal muscular atrophy in infants
Swiss drugmaker Novartis has received US approval for its spinal muscular atrophy gene therapy Zolgensma – pricing the one-time treatment at a record $2.125m.
The Food and Drug Administration on Friday approved Zolgensma for children under the age of two with SMA, including those not yet showing symptoms. The approval covers babies with the deadliest form of the inherited disease as well as those with types where debilitating symptoms may set in later.
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