27 September 2019 - Novartis’ AveXis unit has given its reasons for refusing to supply its spinal muscular atrophy gene therapy Zolgensma to a seriously ill child in Belgium, saying compassionate use rules do no apply as Biogen’s rival Spinraza (nusinersen) is an alternative option.

As pharmaphorum reported earlier this week, this one-off gene therapy for the rare muscle wasting disease is not yet approved in Europe, where it is being reviewed by regulators.

But there is a national campaign to make Zolgensma available to the toddler named as Pia, whose family has raised the cash needed to pay for the drug via crowdfunding.

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