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SMA News & Perspectives

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Daily, digital coverage of the latest SMA news and perspectives.

Daily, digital coverage of the latest SMA news and perspectives.
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Location:

United States

Description:

Daily, digital coverage of the latest SMA news and perspectives.

Language:

English


Episodes

Overview Of The SMA Forums And Recent Discussions & Coming-Of-Age Stories And Living With SMA

10/15/2019
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Forums moderator DeAnn Runge gives us an overview of the forums and updates us on recent discussions. If you haven’t already checked out the forums she encourages you to head on over and introduce yourself. Also, Forums Director Kevin Schaefer talks about how his love of coming-of-age stories relates to living with SMA.

Duration:00:06:24

#44 - Roundtable Discussion - Discussion for October 2019

10/14/2019
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Hosts Kevin Schaefer and Michael Morale discuss the latest news regarding SMA therapies Spinraza and Risdiplam, as well as some of our recent columns. Listen here, and share your thoughts in the SMA News Today forums. For information regarding the DEVOTE trial, please click on the following link: https://www.clinicaltrials.gov/ct2/show/NCT04089566?term=NCT04089566&rank=1 Are you interested in learning more about the latest treatment for spinal muscular atrophy? Visit TreatSMA.com to see how...

Duration:00:28:26

Teen With Epilepsy Described As Rare Case of SMA & Alternative Options For Seating

10/13/2019
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We discuss how clinicians described a rare case of SMA associated with a teen who had epilepsy. Although alternative options for seating look appealing, forums moderator DeAnn Runge explains why this isn’t usually the case for her. She finds her wheelchair to be the most comfortable place. 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...

Duration:00:06:46

Additional Space For Zolgensma Production With AveXis Catalent Partnership & Trending Topics

10/10/2019
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We discuss how additional space for Zolgensma production is the goal of AveXis Catalent partnership and Forums Director Kevin Schaefer talks about some of the trending topics in the SMA News Today forums. 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.

Duration:00:04:09

Problems With Metabolising Fat Molecules Are Worrisome In SMA Children & Being Inspirational

10/8/2019
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SMA News Today’s Director of Multichannel Content, Michael Morale, discusses a study that states that problems with metabolising fat molecules are evident and worrisome in SMA children and Forums Director Kevin Schaefer talks about when people call him “inspirational.”

Duration:00:09:10

Risdiplam Is Raising SMN Levels In Older Patients In Durable Ways & Dealing With Disappointment

10/6/2019
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We discuss early trial data showing how risdiplam is raising SMN levels in older patients in ways that seem durable & Despite having appositive attitude, dealing with disappointment is inevitable. Although it can be difficult, it doesn’t need to be the end of the world. Here are a couple examples of moving past major disappointments. 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...

Duration:00:10:07

Adding Reldesemtiv to Spinraza Improved Muscle Function In An SMA Mouse Model & Finding Humor

10/3/2019
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We discuss how adding Reldesemtiv to Spinraza or the investigational SMN-C1 improved muscle function in an SMA mouse model. Community Development Manager Kevin Schaefer reads an excerpt from Alyssa Silva’s column, “Finding Humor in Stinky Situations.” 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

Duration:00:07:57

Prenatal Blood Test For SMA Is Now Available & "Rolling Through The Seasons"

10/1/2019
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SMA News Today’s Director of Multichannel Content, Michael Morale, discusses how a prenatal blood test for SMA and other single gene diseases is now available. Community Development Manager Kevin Schaefer talks about “rolling through the seasons” when you have SMA.

Duration:00:07:39

#43 - Interview With Jeff Olander

9/30/2019
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In episode 43, host Kevin Schaefer interviews Jeff Olander from Raleigh, North Carolina. Jeff is a PhD student at UNC-Chapel Hill, studying physics. He talks in this interview about his education, career goals, and his life with SMA Type 2.

Duration:00:49:41

Macedonian Gaucher Activist For Rare Disease Patients & Getting Ready For The Cold

9/29/2019
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We discuss a Macedonian Gaucher activist, whose publicizing the plight of rare disease patients. Although fall and winter are her least favorite seasons, DeAnn finds being prepared can help the transition. She shares what she does to welcome, or at least tolerate the change of seasons. 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...

Duration:00:08:29

European Spinraza Label Updated to Reflect Long-Term Benefits & Trending Topics

9/26/2019
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We discuss how the European Spinraza label was updated to reflect long-term benefits in broad ranges of SMA patients and Community Development Manager Kevin Schaefer talks about some of the trending topics in the SMA News Today forums. 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.

Duration:00:07:11

High Chance Of Scoliosis Surgery in Types 1C and 2 & Superheroes With Disabilities

9/24/2019
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SMA News Today’s Director of Multichannel Content, Michael Morale, discusses a Dutch study that states there is a high probability of scoliosis surgery found for SMA types 1C, and 2. Community Development Manager Kevin Schaefer talks about a new memoir by disability rights activist Keah Brown.

Duration:00:08:17

Spinraza Gives Babies With Type 1 SMA Hope For a Healthy Life in North Macedonia

9/22/2019
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We discuss how Spinraza gives babies with type 1 SMA in North Macedonia hope for a healthy life. Also, SMA News Today’s forums moderator, DeAnn Runge, shares how service dogs were introduced into her life, and how service dog fraud jeopardizes their role. 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.

Duration:00:06:57

Phase 2 Trial Shows Presymptomatic Spinraza Treatment to SMA Children Reaching Milestones

9/19/2019
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Community Development Manager Kevin Schaefer talks about some of the trending topics in the SMA News Today forums. We discuss how a phase 2 trial shows presymptomatic Spinraza treatment leads to SMA children reaching otherwise unachievable milestones. 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.

Duration:00:06:51

The World’s First Alport Stamp is a Macedonian Mom’s Latest Win for Rare Disease Patients

9/17/2019
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Community Development Manager Kevin Schaefer talks about some of the trending topics in the SMA News Today forums. SMA News Today’s Director of Multichannel Content, Michael Morale, discusses how the world’s first Alport stamp is a Macedonian mom’s latest win for rare disease patients.

Duration:00:09:04

#42 - Interview With Kyle Harris

9/16/2019
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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

Duration:00:36:27

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

9/15/2019
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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...

Duration:00:08:35

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

9/13/2019
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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.

Duration:00:07:59

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

9/10/2019
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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.

Duration:00:10:03

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

9/8/2019
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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

Duration:00:09:17