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Roche presents new data on risdiplam

  • 6 October 2019

Roche has presented newest data on safety and efficacy of risdiplam, the next drug being developed to treat all forms of spinal muscular atrophy. The data reinforces earlier observations that risdiplam appears to be safe and effective across the full spectrum of the disease.

The company also announced the dates of regulatory submission in the US and EU.

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TreatSMA LIVE – A massive success, thank you all

  • 3 October 2019

We at TreatSMA would love to thank everyone for participating in Saturdays LIVE SMA DAY. We had a whole array of amazing speakers , sports professionals, worldwide known doctors, professionals in SMA and not forgetting those from the community itself. We also had a few visits from the legendary Geordie sausage!

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Hospitals and adult treatments – we need your help

  • 20 September 2019

In a recent update we mentioned that hospitals were going to be asked to submit expressions of interest to provide Spinraza for adult patients. We can now confirm that that request has gone out and it is now down to hospitals to submit to NHS England.

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