SMA News & Perspectives

How SMA-Related Humor Presents Itself in Unexpected Moments, Fundraising Options & Online Therapy

06 May 2020

Contributed by Lukas

Forums Director Kevin Schaefer talks about how SMA-related humor presents itself in unexpected moments. And also, this week the topics included on the...

#57 - Interview with Heather Kerstetter

05 May 2020

Contributed by Lukas

In episode 57, host Kevin Schaefer interviews Heather Kerstetter from Philadelphia, Pennsylvania. Heather is a social worker who has SMA Type 3. She t...

How Taking Part in Adapted Sports Improves Mental Health in Patients With SMA

04 May 2020

Contributed by Lukas

Forums moderator DeAnn Runge discusses how looking at what makes her life easier now determines what would make her life easier moving forward. SMA N...

Discussing The Lack of Muscle Cell Signaling Which is Seen to Kill Motor Neurons in SMA-LED2

01 May 2020

Contributed by Lukas

SMA News Today’s multimedia associate, Price Wooldridge, discusses the lack of muscle cell signaling which is seen to kill motor neurons in SMA-LED2...

Telemedicine, Therapy Platforms, Alternative Exercise Routines & Hospital Preparedness Folders

29 Apr 2020

Contributed by Lukas

Forums Director Kevin Schaefer reads from his latest column about a new at-home exercise routine he’s developed, using his electric ceiling lift. A...

Discussing Motor Gains and Safety with Zolgensma’s IT Use in a STRONG Trial

27 Apr 2020

Contributed by Lukas

While thinking about how to navigate the Coronavirus situation, forums moderator DeAnn Runge draws parallels to a family game they played. SMA News T...

First Patient Treated in The Phase 2/3 Trial, Assessing Higher Doses of Spinraza

24 Apr 2020

Contributed by Lukas

Forums Director Kevin Schaefer writes about a new at-home exercise routine he’s developed, using his electric ceiling lift. SMA News Today’s mult...

Playing Phone Games and Nintendo Switch & Home Exercise Routines and Robotic Assistance Devices

22 Apr 2020

Contributed by Lukas

Forums Director Kevin Schaefer talks about how the pop-culture icon Kevin Smith impacted his life, both as a writer and as a person with a disability....

#56 - SMA Treatments Overview and “Crip Camp” Discussion

21 Apr 2020

Contributed by Lukas

In episode 56, hosts Kevin Schaefer and Michael Morale provide an overview of the different SMA treatments, and discuss recent news about the experime...

How Adult Patients with SMA Benefit From Spinraza Treatments

20 Apr 2020

Contributed by Lukas

To increase her independence, forums moderator DeAnn Runge is debating if she should pursue a service dog, a JACO Robotic Arm or both. Each option pre...

Ideas for Staying Fit and Healthy During Isolation & Approval of Zolgensma for Treating SMA in Japan

17 Apr 2020

Contributed by Lukas

Forums Director Kevin Schaefer talks about ideas for staying fit and healthy during COVID-19 isolation. SMA News Today’s multimedia associate, Pric...

Accessible Toys, Having Fun With Subscription Boxes & Kevin Schaefer's Obsession With Action Figures

15 Apr 2020

Contributed by Lukas

The Easter holiday looked a lot different this year than it traditionally does. Forum members shared their plans for the holiday to make it special. A...

Odd Jobs, Spring Cleaning & French Study Finds Improvements in Palliative Care for SMA Type 1 Babies

13 Apr 2020

Contributed by Lukas

Forums moderator DeAnn Runge is looking for solutions to help with odd jobs and spring cleaning. SMA News Today’s multimedia associate, Price Woold...

FDA Shifts Risdiplam Decision to August to Include Data on Older Patients

10 Apr 2020

Contributed by Lukas

Forums Director Kevin Schaefer talks about the Stephen Hawking biopic “The Theory of Everything,” and how this film explores disability. SMA News...

Managing Spinraza Treatments, Caregivers, Outside Deliveries and More During COVID-19

08 Apr 2020

Contributed by Lukas

COVID-19 is still a popular topic of discussion on the forums. Topics associated include continuing with Spinraza treatments, scheduling caregivers, h...

#55 - Interview With Victor Guerra

07 Apr 2020

Contributed by Lukas

In episode 55, host Kevin Schaefer interviews Victor Guerra from Fresno, California. Victor is an entrepreneur who has SMA Type 2, and he uses his onl...

How Scoliosis Surgery in SMA Children is Linked to Permanent Motor Skill Loss

06 Apr 2020

Contributed by Lukas

Amid the coronavirus crisis forums moderator DeAnn Runge has to decide if she wants to risk exposure to the virus to continue her Spinraza treatments....

Discussing Markers of Inflammation Essential for AAV Gene Therapy Use & Thoughts About “Crip Camp"

03 Apr 2020

Contributed by Lukas

Forums Director Kevin Schaefer talks about the new Netflix documentary “Crip Camp,” which tells the story of a summer camp in the 50s and 60s for ...

The SMA News Today Forums: a Place to Share Ideas That Will Brighten Spirits During Difficult Times

01 Apr 2020

Contributed by Lukas

With a community full of problem solvers the forums have become a place to share ideas that will brighten spirits during these difficult times. Als...

Global Rare Disease Group’s Goal to Have 1,000 New Therapies by 2027 & Benefits of a Service Dog

30 Mar 2020

Contributed by Lukas

Forums moderator DeAnn Runge talks about how having a service dog has enhanced her life. SMA News Today’s multimedia associate, Price Wooldridge, d...

Book, Movie and Spotify Recommendations & Study Recommends a Lower Radiation Dose for Spinraza Scans

27 Mar 2020

Contributed by Lukas

Forums Director Kevin Schaefer shares some book, movie, and Spotify recommendations for people who are on quarantine. SMA News Today’s multimedia a...

Alternative Solutions to Physical Therapy During the COVID-19 Pandemic

25 Mar 2020

Contributed by Lukas

Forums Director Kevin Schaefer talks about alternative solutions to physical therapy. As a result of the COVID-19 pandemic, he has to refrain from goi...

New Test Screens 420 Genes for Markers of SMA & A Message to Friends, Family About COVID-19

23 Mar 2020

Contributed by Lukas

Forums moderator DeAnn Runge shares her message to friends and family in regards to COVID-19 and their lives being turned upside down. SMA News Today...

Tips For Self-Isolation During the Coronavirus & SYT13 Gene Can Prolong Life in Mice With SMA

20 Mar 2020

Contributed by Lukas

Forums Director Kevin Schaefer shares some of his tips for self-isolation during the Coronavirus (COVID-19) outbreak. SMA News Today’s multimedia a...

Coronavirus (COVID-19): Precautions, Protecting Yourself and Staying Productive During Lockdown

18 Mar 2020

Contributed by Lukas

One of the main topics of conversation over on the forums is COVID-19. Posted are links to resources and tips on staying productive during lockdown. ...

#54 - Roundtable About Corona Virus

17 Mar 2020

Contributed by Lukas

In episode 54, Michael and I discuss the global outbreak of Coronavirus (COVID-19). We share our tips for how others in the SMA community can best pro...

Risdiplam Under FDA Review & How the Lack of SMA Protein May Also Directly Contribute to SMA

16 Mar 2020

Contributed by Lukas

Anticipation is growing as Roche/Genentech's treatment risdiplam, the first oral medication to treat SMA, is under FDA review. SMA News Today’s mul...

Importance of Physical Therapy & Institute for Gene Therapies to Modernize Reimbursement Framework

13 Mar 2020

Contributed by Lukas

SMA News Today’s Senior Director of Multichannel Content, Michael Morale, discusses the importance of physical therapy, and how physical therapy can...

COVID19, Spinraza, Bloating, SPC’s, Being Spontaneous & Transferring Out Of Wheelchair To Take a Nap

11 Mar 2020

Contributed by Lukas

In the forums a topic of discussion has been the thing that’s on everyone’s mind, the Novel Coronavirus, also known as COVID19. Spinraza has also...

Being Spontaneous With SMA & How Body Composition May be a Biomarker of Motor Function in SMA

09 Mar 2020

Contributed by Lukas

Spontaneity can be difficult when you have SMA. Sometimes it even takes planning. Forums moderator DeAnn Runge shares how she does it and is looking...

Maintaining Long-Distance Friendships & MDA Executive’s Discussion of Gene Therapies

06 Mar 2020

Contributed by Lukas

Forums Director Kevin Schaefer talks about maintaining long-distance friendships, particularly with people in the SMA community. SMA News Today’s m...

Celebrating Rare Disease Day 2020 with #WhatMakesMeRareSMA & How Music Plays a Big Part in Life

04 Mar 2020

Contributed by Lukas

February 29th was recognized as Rare Disease Day across the world. SMA News Today columnists shared what this day meant to them. A social media camp...

#53 - Interview with Alyssa Silva

03 Mar 2020

Contributed by Lukas

In episode 53, host Kevin Schaefer interviews our very own Alyssa Silva. Alyssa is from Cumberland, Rhode Island, and she is a columnist and forums mo...

Public Interactions with People With Disabilities & Muscular Dystrophy Association Helping Others

02 Mar 2020

Contributed by Lukas

When it comes to public interactions with people who have disabilities there are no set rules. As someone who has a disability, DeAnn shares her thou...

Risdiplam Success in Treating SMA Type 1 Babies in FIREFISH Study & Kevin's Spinraza Injection

28 Feb 2020

Contributed by Lukas

Forums Director Kevin Schaefer talks about his most recent Spinraza injection and SMA News Today’s multimedia associate, Price Wooldridge, discusses...

Talking About Rare Disease Day & Accessible Parking, Sleepovers with SMA and Product Reviews

26 Feb 2020

Contributed by Lukas

Forums Director Kevin Schaefer talks about the upcoming Rare Disease Day, and what it means to him. Also, DeAnn Runge shares the latest activity goin...

The Discovery of a Potential New SMA Genetic Modifier & Having a Great Deal of Strength

24 Feb 2020

Contributed by Lukas

Having gone through a life threatening ordeal, forums moderator DeAnn Runge knows despite being physically weak she has a great deal of strength. As ...

How a CSF Protein Profile May Help Predict Spinraza Responses in Late-Onset SMA Patients

21 Feb 2020

Contributed by Lukas

Forums Director Kevin Schaefer talks about some of the trending topics in the SMA News Today forums. Also, SMA News Today’s multimedia associate, P...

Kevin Schaefer Reads His Week’s Motivational Post & Katie Napiwocki's Comeback as a Columnist

19 Feb 2020

Contributed by Lukas

Forums Director Kevin Schaefer reads from this week’s motivational post in the SMA News Today forums and we're also happy to welcome Katie Napiwocki...

#52 - Getting to Know Luisa Palazola and Rare Disease Day 2020

18 Feb 2020

Contributed by Lukas

In episode 52, host Kevin Schaefer talks with Luisa Palazola, a Community Manager for SMA News Today. Luisa is from Memphis, Tennessee, and she has an...

How Magnetic Rods May Help Scoliosis in Children with SMA & Living With Caution, Not Fear

17 Feb 2020

Contributed by Lukas

Finding the balance between living cautiously and fearfully can be challenging. Forums moderator DeAnn Runge shares how she navigates that delicate b...

Early Access Program From Roche for SMA Therapy Risdiplam in Europe

14 Feb 2020

Contributed by Lukas

SMA News Today’s multimedia associate, Price Wooldridge, discusses an early access program from Roche for SMA therapy Risdiplam in Europe and forums...

Accessible Travel, Becoming Friends With Your Caregivers and The Importance of Exercise With SMA

12 Feb 2020

Contributed by Lukas

Some recent topics being discussed on the forums include accessible travel and becoming friends with your caregivers. Also, Dose of DeAnn has a new ep...

New Treatment Algorithm For SMA Infants Via Vital Newborn Screening & Going To The Bathroom

10 Feb 2020

Contributed by Lukas

SMA News Today’s multimedia associate, Price Wooldridge, discusses a new treatment algorithm for SMA infants diagnosed via vital newborn screening. ...

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

07 Feb 2020

Contributed by Lukas

SMA News Today’s multimedia associate, Price Wooldridge, discusses how the ZPR1 protein may be a new potential therapeutic target for SMA. Also, For...

Embracing The Power of Your Voice & Struggling As a Family

05 Feb 2020

Contributed by Lukas

Forums Director Kevin Schaefer talks about embracing the power of his voice in his latest column. Also, every family goes through their share of trium...

#51 - Interview with Ryan Manriquez and Nora Zade

04 Feb 2020

Contributed by Lukas

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...

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

03 Feb 2020

Contributed by Lukas

SMA News Today’s multimedia associate, Price Wooldridge, discusses how up to 100 free Zolgensma treatments are going to go out worldwide in 2020 und...

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

31 Jan 2020

Contributed by Lukas

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 Monda...

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

29 Jan 2020

Contributed by Lukas

Forums Director Kevin Schaefer talks about trying to manage stomach issues as an individual with SMA & as usual there are great conversations going on...

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

27 Jan 2020

Contributed by Lukas

We discuss a study that suggests that regular CSF exams are urged following Spinraza treatments, to screen for injection side effects. Forums moderato...

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

27 Jan 2020

Contributed by Lukas

We discuss a study that suggests that regular CSF exams are urged following Spinraza treatments, to screen for injection side effects. Forums moderato...

HHS Secretary's Efforts To Cure Rare Diseases & Taking Advantage of New Opportunities

24 Jan 2020

Contributed by Lukas

We discuss the HHS Secretary, Alex Azar, touts the White House efforts to cure rare diseases & Forums Director Kevin Schaefer talks about taking advan...

Being Rare and Resilient & No Topic Is Off Limits on The SMA News Today's Forums

22 Jan 2020

Contributed by Lukas

Go to our forums: http://bit.ly/forumsflashbriefings Forums Director Kevin Schaefer recounts the story of his adventures in Philadelphia, and talks a...

#50 - Roundtable Discussion - January 2020

21 Jan 2020

Contributed by Lukas

In episode 50, hosts Kevin Schaefer and Michael Morale discuss updates on Roche and Genentech’s experimental therapy risdiplam. Now that this drug i...

Spinraza Stabilizes or Improves Motor Function in Older SMA Patients & The Reality of Aging

20 Jan 2020

Contributed by Lukas

We discuss a US real-world study, that claims that Spinraza stabilizes or improves motor function in older SMA patients. Also, when you have a disease...

Multidisciplinary Approach Improves Spinraza Dosing Process in SMA Patients & Parenting and SMA

17 Jan 2020

Contributed by Lukas

We discuss a study that states how a multidisciplinary approach improves the Spinraza dosing process in SMA patients. Also, Forums Director Kevin Scha...

Importance of Media Representation for People With Disabilities & Conversations in the SMA Forums

15 Jan 2020

Contributed by Lukas

Forums Director Kevin Schaefer talks about the importance of media representation for people with disabilities, and calls for more of it in 2020. Also...

Targeted Genetic Screening Helps Diagnosing Patients With SMA-Like Symptoms & Pranking With SMA

13 Jan 2020

Contributed by Lukas

We discuss how targeted genetic screening may be useful in diagnosing patients with SMA-like symptoms. Also, a harmless prank can be fun, but it’s n...

Early Trial Data Shows SRK-015 Increases Myostatin Growth Factor Levels in SMA Patients

10 Jan 2020

Contributed by Lukas

We discuss early trial data that shows SRK-015 increases myostatin growth factor levels up to 100-fold in SMA patients & Forums Director Kevin Schaefe...

Early Trial Data Shows SRK-015 Increases Myostatin Growth Factor Levels in SMA Patients

10 Jan 2020

Contributed by Lukas

We discuss early trial data that shows SRK-015 increases myostatin growth factor levels up to 100-fold in SMA patients & Forums Director Kevin Schaefe...

Volunteering As A Reward & Humorous Interactions With People

08 Jan 2020

Contributed by Lukas

In her most recent column, I Don’t Need to Win to Be a Winner: Volunteering Is Its Own Reward, Halsey explains how her volunteer efforts have been r...

#49 - Interview With Ashley Fox

07 Jan 2020

Contributed by Lukas

In episode 49, host Kevin Schaefer interviews Ashley Fox from Southern California. Ashley is currently studying psychology at a community college, and...

Asuragen States New Screening Kit Analyzes SMN1 and SMN2 Genes in the Lab & Being Grateful

06 Jan 2020

Contributed by Lukas

We discuss how Asuragen is stating that a new screening kit, quickly and thoroughly analyzes SMN1 and SMN2 genes in the lab. & With all the variables ...

AveXis Unaware of the Cause of Inflammation that Led to the STRONG Trial Hold & 2019 Highlights

20 Dec 2019

Contributed by Lukas

We discuss how AveXis is unaware of the cause of inflammation, that led to the STRONG trial hold of AVXS-101. Forums Director Kevin Schaefer shares so...

#48 - Roundtable Discussion December 2019

17 Dec 2019

Contributed by Lukas

In episode 48, hosts Kevin Schaefer and Michael Morale discuss the latest news regarding Roche and Genentech’s experimental therapy risdiplam. We al...

#47 - Interview With James Ian

03 Dec 2019

Contributed by Lukas

In episode 47, host Kevin Schaefer talks with James Ian from Los Angeles, California. James is a singer, songwriter, and actor, and he has SMA Type 3....

#46 Roundtable Discussion - Discussion for November 2019

19 Nov 2019

Contributed by Lukas

In episode 46, hosts Kevin Schaefer and Michael Morale discuss the latest news regarding Roche and Genentech’s experimental therapy Risdiplam. We al...

#45 - Interview With Jim And Becky Berry

05 Nov 2019

Contributed by Lukas

In episode 45, host Kevin Schaefer talks with Jim and Becky Berry from Myrtle Beach, South Carolina. Jim and Becky share their experiences as parents ...

Embracing The Imaginative Spirit of Halloween & SMA News Today Forums

30 Oct 2019

Contributed by Lukas

Forums Director Kevin Schaefer talks about embracing the imaginative spirit of Halloween. Forums moderator DeAnn Runge shares what’s been going on i...

#44 - Roundtable Discussion - Discussion for October 2019

15 Oct 2019

Contributed by Lukas

Hosts Kevin Schaefer and Michael Morale discuss the latest news regarding SMA therapies Spinraza and Risdiplam, as well as some of our recent columns....

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

09 Oct 2019

Contributed by Lukas

SMA News Today’s Director of Multichannel Content, Michael Morale, discusses a study that states that problems with metabolising fat molecules are e...

#43 - Interview With Jeff Olander

01 Oct 2019

Contributed by Lukas

In episode 43, host Kevin Schaefer interviews Jeff Olander from Raleigh, North Carolina. Jeff is a PhD student at UNC-Chapel Hill, studying physics. H...

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

30 Sep 2019

Contributed by Lukas

We discuss a Macedonian Gaucher activist, whose publicizing the plight of rare disease patients. Although fall and winter are her least favorite seaso...

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

27 Sep 2019

Contributed by Lukas

We discuss how the European Spinraza label was updated to reflect long-term benefits in broad ranges of SMA patients and Community Development Manager...

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

25 Sep 2019

Contributed by Lukas

SMA News Today’s Director of Multichannel Content, Michael Morale, discusses a Dutch study that states there is a high probability of scoliosis surg...

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

23 Sep 2019

Contributed by Lukas

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 Rung...

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

20 Sep 2019

Contributed by Lukas

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 show...

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

18 Sep 2019

Contributed by Lukas

Community Development Manager Kevin Schaefer talks about some of the trending topics in the SMA News Today forums. SMA News Today’s Director of Mult...

#42 - Interview With Kyle Harris

17 Sep 2019

Contributed by Lukas

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 access...

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

16 Sep 2019

Contributed by Lukas

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 R...

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

13 Sep 2019

Contributed by Lukas

Community Development Manager Kevin Schaefer reads from a motivational post in the SMA News Today forums. We discuss why screening tests and other ste...

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

11 Sep 2019

Contributed by Lukas

SMA News Today’s Director of Multichannel Content, Michael Morale, discusses an ongoing trial that states how SMA candidate SRK-015 shows promise in...

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

09 Sep 2019

Contributed by Lukas

We discuss a study that suggests children with SMA are at risk of weak bones and fractures and Community Development Manager Kevin Schaefer talks abou...

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

06 Sep 2019

Contributed by Lukas

We discuss how AveXis’ OneGene program could smooth the path for families wanting Zolgensma and Community Development Manager Kevin Schaefer talks a...

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

04 Sep 2019

Contributed by Lukas

SMA News Today’s Director of Multichannel Content, Michael Morale, discusses likely future work into Zolgensma, gene therapies and other SMA treatme...

#41 - Roundtable Discussion - Discussion for September 2019

03 Sep 2019

Contributed by Lukas

Hosts Kevin Schaefer and Michael Morale discuss a recent webinar about AveXis’ gene therapy Zolgensma. Then in part two, they talk about SMA awarene...

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

02 Sep 2019

Contributed by Lukas

We discuss how NORD honors industry in patient advocates at rare impact awards gala and SMA News Today forums moderator DeAnn Runge shares her perspec...

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

30 Aug 2019

Contributed by Lukas

We discuss a study that suggests that suggests Anti-epileptic Keppra could be a candidate for treatment of SMA and Community Development Manager Kevin...

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

28 Aug 2019

Contributed by Lukas

SMA News Today’s Director of Multichannel Content, Michael Morale, discusses how Zolgensma’s arrival adds urgency to SMA newborn screening efforts...

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

26 Aug 2019

Contributed by Lukas

We discuss how Eurordis unveils an integrated care initiative for rare disease patients. Also, SMA News Today’s Director of Multichannel Content, Mi...

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

23 Aug 2019

Contributed by Lukas

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 Sc...

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

21 Aug 2019

Contributed by Lukas

SMA News Today’s Director of Multichannel Content, Michael Morale, discusses an interview where researchers talk about Zolgensma’s price, and its ...

#40 - Interview With Kellie Cusack

20 Aug 2019

Contributed by Lukas

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 currentl...

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

19 Aug 2019

Contributed by Lukas

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 R...

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

16 Aug 2019

Contributed by Lukas

We discuss “Rare Barometer”, which is a program that is helping Eurordis shape EU rare disease policies. Also, Community Development Manager Kevin...

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

14 Aug 2019

Contributed by Lukas

SMA News Today’s Director of Multichannel Content, Michael Morale, discusses how patients with SMA Types 1, 2, & 3, options for Zolgensma exist, and...

Ontario Patients Are Granted With Wider Access To Spinraza & Current Treatments, Brighter Futures

12 Aug 2019

Contributed by Lukas

We discuss how SMA patients in Ontario are granted wider access to Spinraza. Also, SMA News Today’s Director of Multichannel Content, Michael Morale...

Family Says Zolgensma Saved All of Their Lives; Their Gene Therapy & Tyler Dukes

09 Aug 2019

Contributed by Lukas

We discuss a family that states Zolgensma saved all of their lives, and they share their families journey through gene therapy, Also, Community Develo...

Zolgensma's Potential To Change The Whole Landscape of SMA & Melissa Milinovich's Story

07 Aug 2019

Contributed by Lukas

SMA News Today’s Director of Multichannel Content, Michael Morale, discusses how Zolgensma has the potential to change the whole landscape of SMA. A...