form: 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, according to The Independent. SMA is the leading genetic cause of death in infants. 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 disease often leads to paralysis, breathing difficulty and death within months for babies born with the most serious type 1 form. This is potentially a new standard of care for babies with the most serious form of SMA, said Dr Emmanuelle Tiongson, a Los Angeles paediatric neurologist who has provided Zolgensma to patients under an expanded access program. SMA affects about one in every 10,000 live births, with 50% to 70% having type 1.
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