AIFA OKs 1st gene therapy agst SMA

(ANSA) – ROME, MAR 10 – Italian drugs agency AIFA on
Wednesday approved the first gene therapy against Spinal
Muscular Atrophy (SMA), the most common genetic cause of infant
    The drug, Zolgensma, will be available free of charge on the
national health system for all infants under 13.5 kg in weight,
AIFA said.
    The drug will come into use in a single-administration treatment
starting in April.
    The OK came after a process that began last May.
    Some 40-50 children are estimated to be born in Italy each year
with SMA.
    Makers Novartis has also agreed to make the drug freely
available for clinical trials for children weighing between 13.5
and 21 kg. (ANSA).


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