SMA News & Perspectives-logo

SMA News & Perspectives

News & Politics Podcasts >

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

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

Location:

United States

Description:

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

Language:

English


Episodes

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

9/19/2019
More
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
More
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
More
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
More
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
More
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
More
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
More
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

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

9/5/2019
More
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.

Duration:00:09:27

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

9/3/2019
More
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...

Duration:00:12:57

#41 - Roundtable Discussion - Discussion for September 2019

9/2/2019
More
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.

Duration:00:31:28

NORD Honors Industry In Patient Advocates At Rare Impact Awards Gala & Overprotective Parents

9/1/2019
More
We discuss how NORD honors industry in patient advocates at rare impact awards gala and SMA News Today forums moderator DeAnn Runge shares her perspective on overprotective parents. A college friend is coming to visit and instead of allowing her friend, who also has a disability, to do this on her own her mother is tagging along. DeAnn hopes in leading by example her friends Mom will find a better balance between support and independence with her daughter.

Duration:00:08:27

Anti-Epileptic Keppra Can Be A Candidate For SMA Treatment & Batcave For Personal Space

8/29/2019
More
We discuss a study that suggests that suggests Anti-epileptic Keppra could be a candidate for treatment of SMA and Community Development Manager Kevin Schaefer reads his latest column about acquiring personal space, via his Batcave.

Duration:00:08:03

Zolgensma’s Arrival Adds Urgency to SMA Newborn Screening Efforts & Natalie Russo And Her Brother

8/27/2019
More
SMA News Today’s Director of Multichannel Content, Michael Morale, discusses how Zolgensma’s arrival adds urgency to SMA newborn screening efforts in the US and Community Development Manager Kevin Schaefer reads one of the final 31 Days of SMA stories. In this story, Natalie Russo talks about her and her brother having SMA.

Duration:00:09:29

Eurordis' Integrated Care Initiative For Rare Disease Patients & Cooperate With Physical Therapists

8/25/2019
More
We discuss how Eurordis unveils an integrated care initiative for rare disease patients. Also, SMA News Today’s Director of Multichannel Content, Michael Morale, discusses working with physical therapists, and making them more comfortable while working out with patients with 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....

Duration:00:09:04

RNA Molecule miR-23a May Have Therapeutic Role in SMA & From College Writing Course To A Career

8/22/2019
More
We discuss a study that suggests that the small RNA molecule miR-23a, may have a therapeutic role in SMA. Also, Community Development Manager Kevin Schaefer reads from his latest column. In this post, he reflects on how a college writing course prepared him for his career. 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...

Duration:00:08:52

Zolgensma’s Price And Its 'Cure One, Cure Many' Potential & Concerts Accessibility

8/20/2019
More
SMA News Today’s Director of Multichannel Content, Michael Morale, discusses an interview where researchers talk about Zolgensma’s price, and its cure one, cure many potential. Also, Community Development Manager Kevin Schaefer reads from the latest 31 Days of SMA story. This post focuses on concert accessibility. 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...

Duration:00:09:00

#40 - Interview With Kellie Cusack

8/20/2019
More
In episode 40 of the SMA News Today Podcast, host Kevin Schaefer interviews Kellie Cusack. Kellie lives in New Jersey with her family, and is currently studying journalism at a community college. She talks about growing up with SMA Type II, blogging, and her involvement with the SMA community. 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...

Duration:00:28:47

High Levels of Alpha-COP Proteins Seems To Ease SMA Severity in Mouse Models & Spinraza For 2 Years

8/18/2019
More
We discuss how raising levels of Alpha-COP proteins, seems to ease SMA disease severity in mouse models. Also, SMA News Today forums moderator DeAnn Runge shares her perspective about being on Spinraza for 2 years, and also gives an update regarding her 9th Spinraza injection. 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...

Duration:00:07:53

The "Rare Barometer” Is Helping Eurordis Shape EU Rare Disease Policies & SMA Community Diversity

8/15/2019
More
We discuss “Rare Barometer”, which is a program that is helping Eurordis shape EU rare disease policies. Also, Community Development Manager Kevin Schaefer reads from his latest column about diversity within the SMA community. 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:00:09:03

Zolgensma For SMA Types 1, 2 And 3, More Options On The Way & Angela Titcombe

8/13/2019
More
SMA News Today’s Director of Multichannel Content, Michael Morale, discusses how patients with SMA Types 1, 2, & 3, options for Zolgensma exist, and more may be on the way. Also, Community Development Manager Kevin Schaefer reads from Angela Titcombe’s 31 Days of SMA story. Angela talks about being a teacher with 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...

Duration:00:11:07