AveXis rules out EU compassionate use of SMA gene therapy, says Biogen option is available

27 September 2019 - Novartis’ AveXis unit has given its reasons for refusing to supply its spinal muscular atrophy gene ...

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To speed access to compassionate use, look beyond the FDA

10 August 2017 - In mid-July, the nonpartisan research arm of the federal government, the Government Accountability Office, released its long-awaited ...

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What experimental drug? Most companies don’t post compassionate use policies

11 October 2016 - As patients clamour for greater access to experimental medicines, a survey released Tuesday finds that ...

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Statement from FDA Commissioner Robert Califf on the release of the final individual patient expanded access form

2 June 2016 - Today, the U.S. FDA finalized its efforts to streamline the process used by physicians to request expanded ...

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