#103: Clinical Trials and Advocacy with Stephen Mikita and Arya Singh

#103: Clinical Trials and Advocacy with Stephen Mikita and Arya Singh

In episode 103, host Kevin Schaefer talks with Stephen Mikita and Arya Singh, two individuals with SMA who have years of experience with clinical trials. Stephen, 66, was an Assistant Attorney General for the state of Utah for more than 30 years, representing the three largest state agencies providing services and protections for individuals with disabilities. As one of the oldest survivors of SMA, Mr. Mikita has been uniquely positioned to advocate for the patient perspective at every stage of drug development. Arya is a senior at Yale and has dedicated much of her childhood to clinical research. Her parents are the founders of The SMA Foundation, which has brought some of the world’s greatest scientists into SMA research. Arya herself has been a part of clinical research, and this experience was the inspiration behind a children’s book, Courageous Calla & The Clinical Trial. She is a member of the New Haven Commission on Disabilities, with plans to obtain her undergrad degree this spring and a master’s on public health next year. ================================ The SMA Foundation: https://smafoundation.org/ Arya’s book: https://www.amazon.com/Courageous-Calla-Clinical-Trial-Singh/dp/B0892B4D8G ================================ To learn more about Evrysdi, please visit http://www.ApprovedForSMA.com ================================ To take part in our ongoing discussions regarding SMA, please visit http://www.smanewstoday.com/forums ================================ For the most up-to-date information regarding SMA, please visit www.smanewstoday.com

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#42 - Interview With Kyle Harris

#42 - Interview With Kyle Harris

In episode 42, host Kevin Schaefer talks with Kyle Harris from San Diego, California. Kyle talks about growing up with SMA Type 3, college, and accessibility in the modern world. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy, including its history and how it is being investigated for the treatment of genetic diseases. Visit www.exploregenetherapy.com

17 Syys 201936min

RNA Therapy Delivered by Viral Vector May Hold Potential to Treat SMA

RNA Therapy Delivered by Viral Vector May Hold Potential to Treat SMA

We discuss how an RNA therapy that’s delivered by a viral vector may hold potential to treat SMA. Also, SMA News Today’s forums moderator, DeAnn Runge discusses how airlines do not allow wheelchair users to stay in their chairs during the flights. She describes how vulnerable it feels being separated from your chair during airline travel. Are you interested in learning more about the latest treatment for spinal muscular atrophy? Visit TreatSMA.com to see how this treatment works, hear about family stories, and learn about the steps to starting treatment. Visit TreatSMA.com.

16 Syys 20198min

Why Screening Tests For Zolgernsma’s Use Are Necessary & Motivational Post

Why Screening Tests For Zolgernsma’s Use Are Necessary & Motivational Post

Community Development Manager Kevin Schaefer reads from a motivational post in the SMA News Today forums. We discuss why screening tests and other steps for Zolgensma’s use are necessary.

13 Syys 20198min

SMA Candidate SRK-015 Shows Promise in Healthy Volunteers & More Independence

SMA Candidate SRK-015 Shows Promise in Healthy Volunteers & More Independence

SMA News Today’s Director of Multichannel Content, Michael Morale, discusses an ongoing trial that states how SMA candidate SRK-015 shows promise in healthy volunteers and Community Development Manager Kevin Schaefer talks about his journey toward more independence.

11 Syys 201910min

Study Says Children With SMA Are at Risk of Weak Bones And Fractures & More Independence

Study Says Children With SMA Are at Risk of Weak Bones And Fractures & More Independence

We discuss a study that suggests children with SMA are at risk of weak bones and fractures and Community Development Manager Kevin Schaefer talks about his journey toward more independence. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy, including its history and how it is being investigated for the treatment of genetic diseases. Visit www.exploregenetherapy.com

9 Syys 20199min

How AveXis’ OneGene Program Could Help Families Wanting Zolgensma & Trending Topics

How AveXis’ OneGene Program Could Help Families Wanting Zolgensma & Trending Topics

We discuss how AveXis’ OneGene program could smooth the path for families wanting Zolgensma and Community Development Manager Kevin Schaefer talks about some trending discussions in the SMA News Today forums.

6 Syys 20199min

Future Work Into Zolgensma, Gene Therapy And Other SMA Treatments & Physical Books

Future Work Into Zolgensma, Gene Therapy And Other SMA Treatments & Physical Books

SMA News Today’s Director of Multichannel Content, Michael Morale, discusses likely future work into Zolgensma, gene therapies and other SMA treatments. Community Development Manager Kevin Schaefer talks about the nuances of reading a physical book when you have SMA. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy, including its history and how it is being investigated for the treatment of genetic diseases. Visit www.exploregenetherapy.com

4 Syys 201912min

#41 - Roundtable Discussion - Discussion for September 2019

#41 - Roundtable Discussion - Discussion for September 2019

Hosts Kevin Schaefer and Michael Morale discuss a recent webinar about AveXis’ gene therapy Zolgensma. Then in part two, they talk about SMA awareness month, and a couple of our recent columns.

3 Syys 201931min

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