#18 SPECIAL EDITION - AveXis Announcement For FDA Approval Of AVXS101

#18 SPECIAL EDITION - AveXis Announcement For FDA Approval Of AVXS101

In this special edition of the SMA News today podcast, Forums Editor Michael Morale discusses AveXis' recent announcement that experimental SMA gene therapy AVXS-101 has been submitted to the US Food and Drug Administration (FDA) for approval.

Jaksot(443)

ZPR1 Protein May Be New PotentialTherapeutic Target For SMA & NBC's Pilot Featuring Kid With SMA

ZPR1 Protein May Be New PotentialTherapeutic Target For SMA & NBC's Pilot Featuring Kid With SMA

SMA News Today’s multimedia associate, Price Wooldridge, discusses how the ZPR1 protein may be a new potential therapeutic target for SMA. Also, Forums Director Kevin Schaefer talks about an NBC pilot...

7 Helmi 20205min

Embracing The Power of Your Voice & Struggling As a Family

Embracing The Power of Your Voice & Struggling As a Family

Forums Director Kevin Schaefer talks about embracing the power of his voice in his latest column. Also, every family goes through their share of triumphs and tribulations. In Michael Casten’s latest c...

5 Helmi 20205min

#51 - Interview with Ryan Manriquez and Nora Zade

#51 - Interview with Ryan Manriquez and Nora Zade

In episode 51, I talked with Ryan Manriquez and Nora Zade from Sacramento, California. Ryan is a college student with SMA Type 2, and he and Nora have been dating for several months. Both are sophomor...

4 Helmi 202021min

100 Free Zolgensma Treatments Worldwide in 2020 & Unplugging the iPad, Waste of Time

100 Free Zolgensma Treatments Worldwide in 2020 & Unplugging the iPad, Waste of Time

SMA News Today’s multimedia associate, Price Wooldridge, discusses how up to 100 free Zolgensma treatments are going to go out worldwide in 2020 under an access program. Also, one thing DeAnn Runge st...

3 Helmi 20208min

Adults With SMA Tend To Report Low Prevalence of Non-Motor Symptoms & Reading From the SMA Forums

Adults With SMA Tend To Report Low Prevalence of Non-Motor Symptoms & Reading From the SMA Forums

We discuss a study that finds adults with SMA, tend to report low prevalence of non-motor symptoms & Forums Director Kevin Schaefer reads from a Monday Motivation post in the SMA News Today Forums. A...

31 Tammi 20205min

Managing Stomach Issues As SMA Patient & Great Conversation in the SMA News Today Forums

Managing Stomach Issues As SMA Patient & Great Conversation in the SMA News Today Forums

Forums Director Kevin Schaefer talks about trying to manage stomach issues as an individual with SMA & as usual there are great conversations going on in the SMA News Today forums, says DeAnn Runge. D...

29 Tammi 20205min

Regular CSF Exams Are Urged Following Spinraza Treatments & Smart Home Devices

Regular CSF Exams Are Urged Following Spinraza Treatments & Smart Home Devices

We discuss a study that suggests that regular CSF exams are urged following Spinraza treatments, to screen for injection side effects. Forums moderator DeAnn Runge has an affinity for smart home devic...

27 Tammi 20208min

Regular CSF Exams Are Urged Following Spinraza Treatments & Smart Home Devices

Regular CSF Exams Are Urged Following Spinraza Treatments & Smart Home Devices

We discuss a study that suggests that regular CSF exams are urged following Spinraza treatments, to screen for injection side effects. Forums moderator DeAnn Runge has an affinity for smart home devic...

27 Tammi 20208min

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