Will NIH And NICE Recommend Funding of Spinraza in England? & Each Day As A Blessing
12m Jul 22, 2019
12m
Mom of a Child Who Lived Through Death With SMA and Through Life With Spinraza
9m Jul 19, 2019
9m
Zolgensma Helps Babies Treated Early But Older Patients May Also Benefit & 2019 Cure SMA
15m Jul 17, 2019
15m
#38 - Roundtable Discussion - Discussion for July 2019
In the latest episode of the SMA News Today Podcast, hosts Kevin Schaefer and Michael Morale discuss updates regarding SMA therapies Spinraza and Reldesemtiv. Also, Kevin and columnist Brianna Albers share their experiences at the 2019 Cure SMA conference. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy, including its history ...
40m Jul 16, 2019
40m
NCATS Warns That Rare Diseases Constitute Public Health Issue & Going With The Flow
We discuss how the National Center For Advancing Translational Studies, (NCATS), warns that rare diseases constitute a public health issue. Also, SMA News Today forums moderator DeAnn Runge shares her perspective about going with the flow and living with SMA. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy, including its histo...
8m Jul 15, 2019
8m
Study Suggests Spinraza Is Safe And Effective In Adults With SMA & Negative Experiences With Flying
We discuss a recent study that suggests that Spinraza is safe and effective in adults with SMA. Also, Community Development Manager Kevin Schaefer reads from his latest column, in which he recounts a negative experience with flying. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy, including its history and how it is being inve...
9m Jul 12, 2019
9m
Rare Case of Severe SMA in India & The Cure SMA Conference 2019
SMA News Today’s Director of Multichannel Content, Michael Morale, discusses a rare case of severe SMA in India, that was partly evident by bone fractures at birth. Also, Community Development Manager Kevin Schaefer talks about his experiences at the 2019 Cure SMA conference. Cure SMA is the largest SMA nonprofit organization, and this year’s conference took place in Anaheim, California. Are y...
12m Jul 10, 2019
12m
Risdiplam Is Showing Promise For Treating SMA Types 2 And 3 & Airline Companies And The FAA Updates
We discuss how Risdiplam is showing promise for treating SMA Types 2 and 3. Also, SMA News Today’s Director of Multichannel Content, Michael Morale, discusses how airline companies, and the FAA, refuse to make it easier for those of us in wheelchairs to have the ability to fly like the able-bodied. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information...
12m Jul 08, 2019
12m
Risdiplam Improves Survival and Motor Functions in Infants With Type 1 SMA & The 9th Birthday
We discuss how Risdiplam improves survival and motor functions in infants with Type 1 SMA. Also, Columnist Michael Casten reflects on his daughter's ninth birthday, and how far she's come since her initial SMA diagnosis. 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...
9m Jul 05, 2019
9m
SBMA Patients More Likely To Be Affected by Metabolic Disorders & Going To Faraway Coffee Shops
SMA News Today’s Director of Multichannel Content, Michael Morale, discusses a study that states that SBMA patients are more likely to be affected by metabolic disorders, including heart and liver disease. Also, Columnist Alyssa Silva writes about how spontaneous adventures to faraway coffee shops help her celebrate the good days with SMA. Are you interested in understanding gene therapy? Expl...
8m Jul 03, 2019
8m
#37 - Interview With Courtney Smith And Trevor Rogerson
In episode 37 of the SMA News Today Podcast, host Kevin Schaefer interviews Courtney Smith and her boyfriend Trevor Rogerson. They live in Seattle, Washington, and Courtney has SMA Type 2. 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 diseas...
21m Jul 02, 2019
21m
Zolgensma Extends Survival in SMA Type 1 Patients Years After Dosing & How Will SMA Change
We discuss how Zolgensma continues to extend survival in SMA Type 1 patients several years after dosing. Also, SMA News Today forums moderator DeAnn Runge shares her perspective with regards to how she sees SMA changing in the future, for those of us who suffer from this disease. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy...
10m Jul 01, 2019
10m
SMA Patients in Saskatchewan, Canada, Gained Wider Access to Spinraza & Recognizing Limitations
We discuss how SMA patients in Saskatchewan, Canada, have gained wider access to Spinraza. Also, Community Development Manager Kevin Schaefer talks about recognizing his limitations in his latest column. 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...
7m Jun 28, 2019
7m
Spinraza Restores Motor Function in Children With Late Onset SMA & Kevan Chandler's Book
SMA News Today’s Director of Multichannel Content, Michael Morale, discusses a long-term trial that states how Spinraza was found to improve and restore motor function in children with late onset SMA. Also, Community Development Manager Kevin Schaefer talks about Kevan Chandler's latest book, which is in stores now. Chandler is an author and nonprofit founder, who has SMA. Are you interested i...
9m Jun 26, 2019
9m
High Intensity Training Improves Fitness While Safe for SBMA Patients & Tips for Spinraza Treatments
We discuss a study that suggests how high intensity training improves fitness while being safe for patients with spinal bulbar muscular atrophy (SBMA). Also, SMA News Today’s Director of Multichannel Content, Michael Morale, discusses 4 tips that can make your upcoming Spinraza treatments easier. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information a...
11m Jun 24, 2019
11m
Study Suggests Parents Need Supportive Care With Children Transitioning To Wheelchairs & Spinraza
We discuss a recent study that suggests how parents need supportive care as their children transition to wheelchairs. Also, Community Development Manager Kevin Schaefer talks about how Spinraza has affected his head and neck control in his latest column. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy, including its history an...
9m Jun 21, 2019
9m
Spinraza Users Share Stories in Biogen's Webcast & Swimming Pool Lifts and Disability
SMA News Today’s Director of Multichannel Content, Michael Morale, discusses how some Spinraza users shared their stories in a Biogen webcast on how they went from a gut wrenching diagnosis to a wonderful drug. Also, Community Development Manager Kevin Schaefer reads from a forum topic about swimming pool lifts for people with disabilities. Are you interested in understanding gene therapy? Exp...
9m Jun 19, 2019
9m
#36 - Roundtable Discussion - Discussion for June 2019
In episode 36 of the SMA News Today Podcast, hosts Kevin Schaefer and Michael Morale discuss the FDA-approval of AveXis’ drug Zolgensma, and related news stories. We also look at some of our recent columns and forum posts, and share our thoughts. Listen here, and be sure to subscribe to the podcast. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful informatio...
35m Jun 18, 2019
35m
Nonprofit Group Raising Awareness for Rare Diseases in India & SMA Teaching Patience
We discuss how a nonprofit group is working to raise rare disease awareness in India. Also, SMA News Today forums moderator DeAnn Runge shares her perspective regarding how living with SMA has taught her the meaning of patience. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy, including its history and how it is being investig...
11m Jun 17, 2019
11m
Study on the Underlying Cause of Motor Neuron Degeneration in SBMA & Getting Blood Drawn
We discuss a recent mouse study that explores the underlying cause of motor neuron degeneration in SBMA. Also, Community Development Manager Kevin Schaefer talks about finding the sweet spot, when it comes to getting blood drawn and positioning in his wheelchair. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy, including its h...
9m Jun 14, 2019
9m
PALS Is Offering Free Flights To Rare Diseases Patients & Head And Neck Control With Spinraza
SMA News Today’s Director of Multichannel Content, Michael Morale, discusses how Patient Airlift Services (PALS) uplifts the spirits of rare disease patients by offering free flights. Also, Community Development Manager Kevin Schaefer talks about how his head and neck control have improved since he started receiving Spinraza. Are you interested in understanding gene therapy? ExploreGeneTherapy...
7m Jun 12, 2019
7m
Zolgensma Is Helping Babies To Breathe And Sit On Their Own & Keep Improving Your Life
We discuss how ZOLGENSMA is helping treated babies to breathe and sit on their own. Also, SMA News Today’s Director of Multichannel Content, Michael Morale, discusses how his best friend inspired him to continue to improve and enrich his life. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy, including its history and how it is...
10m Jun 10, 2019
10m
Changes in Bladder and the Colon May Underlie Gastrointestinal Issues in SMA & Medical Advancements
We discuss a recent study that suggests how certain changes in bladder and the colon, may underlie gastrointestinal issues in SMA. Also, Community Development Manager Kevin Schaefer reads from his latest column about what medical advancements mean for adults with SMA. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy, including ...
8m Jun 07, 2019
8m
Early Non-Neuromuscular Symptoms And The Lack of SMN Protein & Zolgensma Versus Spinraza
SMA News Today’s Director of Multichannel Content, Michael Morale, discusses a potential relationship that was found between early non-neuromuscular symptoms and the lack of SMN protein in SMA patients. Also, Community Development Manager Kevin Schaefer reads from a recent post in the SMA News Today forum regarding the recent FDA-approval of Avexis' Zolgensma, and we explain the key differences...
9m Jun 05, 2019
9m
#35 - Interview With Mike Blakey
In episode 35 of the SMA News Today Podcast, host Kevin Schaefer interviews Mike Blakey from Gainesville, Florida. Mike is a part-time CPA and attorney, and he has SMA Type 3." Photo Contributed by Mike Blakey 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 treat...
36m Jun 04, 2019
36m
ACTIVE Game Makes Monitoring Motor Function Fun & Life Expectancy
We discuss how a videogame by the name of ACTIVE, makes it more fun for patients to monitor their motor function. Also, SMA News Today forums moderator DeAnn Runge, shares her perspective regarding life expectancy while living with spinal muscular atrophy. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy, including its history ...
8m Jun 03, 2019
8m
SMA Patients' Mothers Feel Better After Engaging In Social Activities & Home Alone With SMA
We discuss how mothers of SMA patients feel better after engaging in social and leisure activities. Also, Community Development Manager Kevin Schaefer reads from his latest column, about staying home alone 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...
8m May 31, 2019
8m
Saudi Arabian Doctors Need To Be More Aware of Guidelines & Benefits of Eye Tracking Tech
SMA News Today’s Director of Multichannel Content, Michael Morale, discusses how Saudi Arabian doctors need to be more aware of guidelines and new care for SMA. Also, Community Development Manager Kevin Schaefer reads from a recent post in the SMA News Today forums that describes the benefits of eye tracking technology for people with disabilities. Are you interested in understanding gene ther...
7m May 29, 2019
7m
ZOLGENSMA Is The 2nd FDA Approved Treatment For SMA & Speech Generating Devices For Children
SMA News Today’s Director of Multichannel Content, Michael Morale, reads from an article published on the SMA News Today website regarding the official approval of ZOLGENSMA as the 2nd FDA approved treatment for spinal muscular atrophy. Also, we discuss a parent survey that showed how speech generating devices provide benefits for children with SMA Type 1. Link to article regarding FDA approva...
17m May 27, 2019
17m
From Pediatric to Adult Care for SMA Patients & Getting an Accessible Hotel Room
We discuss how a study highlighted the difficulties of transitioning from pediatric to adult care for SMA patients. Also, Community Development Manager Kevin Schaefer reads from his latest column, in which he describes the challenges of acquiring an accessible hotel room. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy, includ...
10m May 24, 2019
10m
Type 2 SMA Patients Are Doing Well With Zolgensma Regarding Motor Milestones & Home Alone With SMA
SMA News Today’s Director of Multichannel Content, Michael Morale, discusses how well Type 2 SMA patients are doing regarding motor milestones and safety concerns regarding taking Zolgensma. Also, Community Development Manager Kevin Schaefer reads from a forum topic about staying home alone when you have SMA. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful ...
11m May 22, 2019
11m
#34 - Roundtable Discussion - Discussion for May 2019
In episode 34 of the SMA News Today Podcast, hosts Kevin Schaefer and Michael Morale talk about the latest SMA news and perspectives from May 2019. Topics include clinical trial updates from the 2019 American Academy of Neurology (AAN) Annual Meeting, as well as some of our recent forum posts and columns. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful info...
28m May 21, 2019
28m
Protease Inhibitors Show Potential to Treat SMA by Raising SMN Levels & Physical and Inner Strength
We discuss how Protease Inhibitors show potential to treat SMA by raising SMN levels. Also, SMA News Today forums moderator DeAnn Runge shares her perspective regarding not only physical strength, but inner strength as well. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy, including its history and how it is being investigated...
9m May 20, 2019
9m
Upper Survival Rate For Adults And Children With Neuromuscular Disorders & Being Kind
We discuss how the survival rate is up for adults and children with neuromuscular disorders. Also, SMA News Today’s Director of Multichannel Content, Michael Morale, discusses how showing respect and being kind, can improve not only your own life, but the lives of others. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy, includ...
9m May 17, 2019
9m
Study on How Leuprorelin Acetate Reduces Swallowing Dysfunctions in SBMA & Unique Interactions
SMA News Today’s Director of Multichannel Content, Michael Morale, discusses a study that finds how Leuprorelin Acetate may reduce swallowing dysfunctions in SBMA patients. Also, Community Development Manager Kevin Schaefer reads from his latest column, in which he describes how having SMA has sparked unique interactions with people. Are you interested in understanding gene therapy? ExploreGen...
10m May 15, 2019
10m
MDA Conference Brought Neuromuscular Experts Together & What To Ask About Zolgensma?
We discuss how the MDA conference brought neuromuscular disease experts together to share a new era in treatments. Also, Community Development Manager Kevin Schaefer talks about what questions adults with SMA should ask in regards to Zolgensma. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy, including its history and how it i...
8m May 13, 2019
8m
Better Screening Techniques Improved Advancements in SMA Treatments & Knowing Your Limitations
We discuss how better screening techniques have improved advancements in SMA treatments. Also, SMA News Today forums moderator DeAnn Runge, shares her perspective regarding knowing your limitations while living with SMA, and not letting these limitations dictate how you live. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy, in...
9m May 10, 2019
9m
Brexit Can Have Real Effects For UK Rare Disease Patients & Loving Physical Therapy
SMA News Today’s Director of Multichannel Content, Michael Morale, discusses how Brexit could have real effects for UK rare disease patients. Also, Community Development Manager Kevin Schaefer reads from his latest column, in which he talks about how he’s come to love physical therapy. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene t...
13m May 08, 2019
13m
Valproic Acid Improves Gross Motor Function, Not Respiratory Function & New Zolgensma Forum
We discuss how Valproic Acid improves gross motor function, but not respiratory function in SMA patients. Also, Community Development Manager Kevin Schaefer introduces a forum dedicated to Zolgensma, a gene therapy designed to treat SMA. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy, including its history and how it is being...
7m May 06, 2019
7m
#33 - Interview With John Scurto
In episode 33 of the SMA News Today Podcast, host Kevin Schaefer interviews John Scurto from Boca Raton, Florida. John is a senior at Florida Atlantic University, studying business management, and he has SMA Type II.” Photo Contributed by John Scurto Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy, including its history and h...
30m May 06, 2019
30m
Cure SMA And PPMD Join Prometheus Research in Data Partnership & The Right Aid For Your Kid
We discuss how Cure SMA and PPMD join Prometheus Research in a data collection partnership. Also, Community Development Manager Kevin Schaefer shares tips for parents about finding the right aid for their child 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...
9m May 03, 2019
9m
Spinraza Could Help With Skin Lesions & Tips On Finding Aids For Children With SMA
SMA News Today’s Director of Multichannel Content, Michael Morale, discusses how skin lesions in an infant with SMA Type 1, were cured after their Spinraza treatment. Also, Community Development Manager Kevin Schaefer shares tips for parents about finding the right aid for their child with SMA. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information abo...
9m May 01, 2019
9m
SMA Newborn Screening Expands With Mandatory Screening & Our Decisions Affect Those Around Us
We discuss how SMA newborn screening is expanding as more states enact mandatory screening. Also, SMA News Today’s Director of Multichannel Content, Michael Morale, discusses how important it is to remember how our decisions affect those who live around us. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy, including its history...
11m Apr 29, 2019
11m
Why SMA Patients Don’t Need Higher Radiation Doses in CT-Guided Spinraza Injections & New Column!
We discuss a study that explains why SMA patients don’t need higher radiation doses in CT-guided Spinraza injections. Also, Community Editor Kevin Schaefer introduces a new column by Katie Napiwocki called “A Wildflower in the Wheelderness.” Kevin reads an excerpt from this column, and invites listeners to check it out on SMA News Today. Are you interested in understanding gene therapy? Explor...
7m Apr 25, 2019
7m
How Moral Dilemmas Complicate Treatment of Rare Diseases & Making Big Occasions Inclusive
SMA News Today’s Director of Multichannel Content, Michael Morale, discusses how moral dilemmas complicate treatment of rare diseases. Also, Community Editor Kevin Schaefer reads from his latest column, in which he describes making big occasions inclusive for people with disabilities. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene th...
10m Apr 24, 2019
10m
New Endurance Test Can Measure Fatigue in Types 2, 3, & 4 SMA Patients & Accomplishing Goals
We discuss how a new endurance test can measure fatigue in types 2, 3, & 4 SMA patients. Also, SMA News Today forums moderator DeAnn Runge shares her perspective on accomplishing your goals while living 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 tre...
10m Apr 22, 2019
10m
Britain Pushes To Make Spinraza Available & Travel and Socializing With SMA
We discuss how the British MP is pushing to make Spinraza available for SMA patients. Also, Community Editor Kevin Schaefer reads from his latest column, in which he describes how SMA has affected the way he travels and socializes. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy, including its history and how it is being inves...
9m Apr 18, 2019
9m
#32 - Roundtable Discussion - Discussion for April 2019
In episode 32 of the SMA News Today Podcast, Community Editor Kevin Schaefer and Multichannel Content Director Michael Morale talk about the top news stories from April 2019. This episode covers newborn screenings, an experimental video game that could test the motor function in people with SMA, and some of our recent columns and forum posts. Listen to the episode here, and be sure to subscrib...
29m Apr 17, 2019
29m
Phase 2 Trial Planned for SRK-015 After Positive Phase 1 & On-Land Versus Aquatic Therapy
We discuss how a phase 2 trial is planned for SRK-015 in SMA patients after positive phase 1 data was reported. Also, Community Editor Kevin Schaefer talks about trying on-land physical therapy, and how it compares to his usual aquatic therapy. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy, including its history and how it i...
10m Apr 17, 2019
10m
Spinraza and ZOLGENSMA Improve Lives of Kids With SMA & Accomplishing Your Goals
We discuss how Spinraza and ZOLGENSMA both dramatically improve the lives of kids with SMA. Also, SMA News Today forums moderator DeAnn Runge, shares her perspective on accomplishing your goals while living 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...
5m Apr 15, 2019
5m