Last Week’s Webinar on Treatment Access and Clinical Trials Now Available

A recording of last week’s webinar updating the community on treatment access and clinical trials is now available online. A PDF of the webinar presentation is also available for download.

An update on the status of Spinraza dosing across the US included the following information:

  • As of December 2017, nearly 2,000 US patients had been dosed.
  • 215 sites have dosed at least one patient, with an average of 9 dosed patients per site.
  • 118 sites have been confirmed on Cure SMA’s site list.
  • 35 states plus DC have at least one site on the Cure SMA list.

We also announced that resources for the AveXis, Genentech/Roche and Biogen drug programs are now available on our website, as these programs have reached pivotal trial stage, or have been approved by the FDA.

Additionally, a list of current and upcoming clinical trials was included in the webinar. The webinar also discussed the importance of developing combination therapies for SMA, and considerations that each family should evaluate when choosing between an approved therapy or participation in a clinical trial.

View the webinar 

Download the presentation 

For more information about Spinraza, visit

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