Calculating SMN1 Copies & Identifying SMA Carriers & Spinal Fusion Surgery Benefits
We discuss how scientists have created a new method to calculate the number of SMN1 copies and identify SMA carriers. Also, Community Editor Kevin Schaefer talks about his spinal fusion surgery in 2005, and why this is an important procedure for many people who have SMA. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy, includi...
8m Apr 12, 2019
8m
Parents Want Better Communications With Healthcare Staff & Absurd Humor
SMA News Today’s Director of Multichannel Content, Michael Morale, discusses how parents want better communications with health care staff regarding their children with SMA. Also, Community Editor Kevin Schaefer reads from his latest column, in which he talks about how absurd humor has helped him cope with the challenges of SMA over the years. Are you interested in understanding gene therapy? ...
8m Apr 10, 2019
8m
WODC 2019 Will Receive 1200 Attendees & Physical Therapy Improves Recovery Efforts
We discuss how organizers expect 1,200 people to attend the Rare Disease Conference known as WODC 2019 in April. Also, SMA News Today’s Director of Multichannel Content, Michael Morale, discusses how physical therapy has improved his recovery efforts when he’s sick. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy, including it...
9m Apr 08, 2019
9m
#31 - Interview With Angela And Justin Titcombe
In episode 31 of the SMA News Today Podcast, Community Editor Kevin Schaefer interviews Angela and Justin Titcombe from Houston, Texas. Angela is a schoolteacher who has SMA type 2, and she and Justin got married in June of 2018. During this interview, the couple shares their story, and talks about what it’s like to be an interabled couple in today’s society. Are you interested in understandin...
38m Apr 08, 2019
38m
Spinraza for Most SMA Patients in China & Ben Mattlin's Book on Inter-abled Relationships
We discuss how China has approved Spinraza for most SMA patients. Also, Community Editor Kevin Schaefer reads from his review of a book about inter-abled relationships by Ben Mattlin. Mattlin is a Los Angeles-based writer with SMA Type 2, and this review is available on our main website and on our forums. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful info...
11m Apr 05, 2019
11m
FDA Revising Guidelines on Developing Treatments for Rare Diseases & “Dr. Phil's" Inter-Abled Couple
SMA News Today’s Director of Multichannel Content, Michael Morale, discusses how the FDA is revising the draft guidelines on developing treatments for rare diseases. Also, Community Editor Kevin Schaefer reads from his latest column, in which he addresses a recent episode of “Dr. Phil” that featured an inter-abled couple. Are you interested in understanding gene therapy? ExploreGeneTherapy.com...
10m Apr 03, 2019
10m
New Gene Therapy is Able to Repair Mutations Causing SMA & Coping With Getting Older
We discuss how a new gene therapy approach is able to repair mutations causing SMA, and other inherited diseases. Also, SMA News Today’s Director of Multichannel Content, Michael Morale, discusses how he copes with getting older. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy, including its history and how it is being investi...
10m Apr 01, 2019
10m
NIH Rare Disease Day & Personal Aides at School as Preparation for Caregivers as an Adult
We discuss how the NIH Rare Disease Day highlights joint networks advancing array of research. Also, Community Editor Kevin Schaefer reads from his latest column, in which he talks about how having personal aides with him in school prepared him for having caregivers as an adult. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy,...
10m Mar 29, 2019
10m
Healthcare for Children With SMA Needs Improvement & Living With Permanent Feeding Tube
SMA News Today’s Director of Multichannel Content, Michael Morale, discusses how parents believe healthcare for children with SMA needs to be improved. Also, SMA News Today forums moderator DeAnn Runge, shares her perspective on living with a permanent feeding tube while living with SMA. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene...
10m Mar 27, 2019
10m
SMA Patients from Ireland and UK Demanding Reimbursement for Spinraza & Humor as Key
We discuss how SMA patients in Ireland and the UK are demanding reimbursement for the cost of Spinraza. Also, SMA News Today’s Director of Multichannel Content, Michael Morale, discusses how humor has played a key role in his life. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy, including its history and how it is being inves...
14m Mar 25, 2019
14m
#30 - Roundtable Discussion - Featured Articles
In episode 30 of the SMA News Today Podcast, Kevin Schaefer and Michael Morale discuss the latest news regarding Spinraza (nusinersen) and Zolgensma (AVXS-101). Both of these drugs are designed to treat SMA. Also, Kevin and Michael take a look at some of the trending discussion topics on the SMA News Today forums, as well as a recent column by Brianna Albers. Are you interested in understandi...
30m Mar 25, 2019
30m
Spinraza's Developers Disappointed With Canadian Agency & SMA as Part of Self Identity
We discuss how the developers of Spinraza are disappointed with a Canadian agency’s updated recommendation. Also, Community Editor Kevin Schaefer reads from his latest column, in which he talks about SMA as a part of his identity. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy, including its history and how it is being invest...
9m Mar 22, 2019
9m
3D Cell Model Mimics SMA Motor Neurons & How to Sign Up SMA News Today Forums
SMA News Today’s Director of Multichannel Content, Michael Morale, discusses how a 3-D cell model mimics SMA motor neurons, and how this is a good platform for testing possible therapies. Also, Community Editor Kevin Schaefer invites listeners to join the SMA News Today forums, and explains how to sign up. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful inf...
10m Mar 20, 2019
10m
Vienna Will Host the RARE2019 Meeting & Michael's 8th Spinraza Injection
We discuss how Vienna is scheduled to host the RARE2019 meeting on rare diseases. Also, SMA News Today’s Director of Multichannel Content, Michael Morale, discusses the results of his 8th Spinraza injection that occurred on Monday, March 11, 2019. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy, including its history and how i...
13m Mar 18, 2019
13m
Spinraza's Cost Effectiveness Exceeds Swedish Willingness to Pay Threshold & Being a Writer With SMA
We discuss how the cost effectiveness of Spinraza exceeds Swedish willingness to pay threshold. Also, Community Editor Kevin Schaefer reads from one of his forum posts, in which he shares advice about being a freelance writer 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 i...
8m Mar 15, 2019
8m
New Technique MUNIX May Capture Motor Neuron Loss and Muscle Status & Kevin's Roadtrip
SMA News Today’s Director of Multichannel Content, Michael Morale, discusses how a new technique called MUNIX may capture motor neuron loss and muscle status in SMA type 3 and 4 adults. Also, Community Editor Kevin Schaefer reads from his latest column, in which he describes a recent road trip he took with friends. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has he...
10m Mar 13, 2019
10m
New Testing Method Called RULM & DeAnn's Journey With Spinraza
We discuss how a testing method known as RULM, can assess the arms’ function in a wider spectrum of SMA patients. Also, SMA News Today forums moderator DeAnn Runge shares her journey, while she goes through her Spinraza treatments. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy, including its history and how it is being inves...
11m Mar 11, 2019
11m
#29 Interview With Kelly Miller
In episode 29 of the SMA News Today Podcast, Community Editor Kevin Schaefer interviews Kelly Miller. Kelly is a retired disability claims representative, and she has SMA type 2. She currently lives in Houston, Texas with her husband. During this interview, Kelly discusses growing up with SMA, going to college away from home, and her marriage. Listeners can connect with Kelly on the SMA News To...
1hr 8m Mar 11, 2019
1hr 8m
ArcherDX Enters Newborn Screening Market & Self-Care for Parents and Caregivers
We discuss how ArcherDX enters the newborn screening market with the acquisition of a company by the name of Baby Gene. Also, Community Editor Kevin Schaefer reads from a forum post about self-care for parents and caregivers in the SMA community. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy, including its history and how it...
6m Mar 08, 2019
6m
SMA and ALS are Linked Via Molecule Crucial to Protein Development & Experiences With PFTs
SMA News Today’s Director of Multichannel Content, Michael Morale, discusses how SMA and ALS are linked via a central hub molecule crucial to protein development. Also, Community Editor Kevin Schaefer reads from his latest column, in which he shares his experiences with pulmonary function tests (PFTs). Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful informa...
9m Mar 06, 2019
9m
Clinical Compound Improves Outcomes in SMA Mouse Models & Michael Talks About His Spinraza Injection
We discuss how a new compound improves clinical outcomes in mouse models of SMA. Also, SMA News Today’s Director of Multichannel Content, Michael Morale, shares his perspectives on his upcoming maintenance dose injection of Spinraza. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy, including its history and how it is being inv...
10m Mar 04, 2019
10m
A Sense of Well-Being & SMA Parents Skeptical About Spinraza
We discuss how a sense of well-being tied to ability, take part in life and engagement with SMA patients. Also, Community Editor Kevin Schaefer reads from a post in the SMA News Today forums. This post provides advice for SMA parents whose kids are skeptical about taking Spinraza. Link to Kevin Schaefer's article: https://smanewstoday.com/forums/forums/topic/when-your-child-with-sma-doesnt-wa...
9m Mar 01, 2019
9m
Cure SMA Campaign Promotes Early Diagnosis and Treatment & Impact of Technology
SMA News Today’s Director of Multichannel Content, Michael Morale, discusses how the Cure SMA campaign promotes early diagnosis and treatment. Also, Community Editor Kevin Schaefer reads from his latest column, in which he talks about the impact of technology in his everyday life. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therap...
7m Feb 27, 2019
7m
#28 Roundtable Discussion - Going To College If You Have SMA
In episode 28 of the SMA News Today Podcast, Community Editor Kevin Schaefer and Multichannel Content Director Michael Morale talk about their college experiences. During this discussion, Kevin and Michael share their perspectives on campus accessibility, independence, and what advice they have for other college students with SMA. Are you interested in understanding gene therapy? ExploreGeneTh...
34m Feb 25, 2019
34m
The Upcoming Rare Disease Day & Sympathy Versus Empathy
We discuss how patients, families and friends will take part in a variety of rare disease day events worldwide on February 28, 2019. Also, SMA News Today’s Director of Multichannel Content, Michael Morale, shares his perspectives on the differences between sympathy versus empathy. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therap...
13m Feb 25, 2019
13m
New SMArtCARE Online SMA Registry & Having Long and Short-Term Goals for Yourself
We discuss how a new SMArtCARE online SMA registry will gather patient data. Also, Community Editor Kevin Schaefer reads from a topic in the SMA News Today forums, in which he talks about having long and short-term goals for himself. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy, including its history and how it is being inv...
7m Feb 22, 2019
7m
Risdiplam Granted With EMA’s PRIME Designation & Letting Children With SMA Have Big Dreams
SMA News Today’s Director of Multichannel Content, Michael Morale, discusses how Risdiplam was granted the EMA’s PRIME designation for potential in SMA. Also, Community Editor Kevin Schaefer reads from his latest column, in which he encourages parents to let their children with SMA have big dreams. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information...
9m Feb 20, 2019
9m
Pennsylvania Clinic for Amish and Mennonite Children With SMA & Scotland's New Rules for Spinraza
We discuss a rural Pennsylvania clinic that treats Amish and Mennonite children who have SMA. Also, SMA News Today’s Multichannel Content Director, Michael Morale, reads an article about how Scotland has opened Spinraza to all SMA patients using its public health system. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy, includi...
12m Feb 18, 2019
12m
New Screening Process May Speed SMN2 Splicing & Talking About Valentine's Day
We discuss how a new screening process may speed work on SMN2 splicing approaches to treat SMA. Also, Community Editor Kevin Schaefer reads from his latest column, in which he shares his perspective on Valentine’s Day. 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 t...
10m Feb 15, 2019
10m
FDA Revises Guidance Regarding Rare Diseases & Being Able to Sit Unassisted With Aquatic Therapy
Michael Morale discusses how the FDA is revising the draft guidance on developing treatments for rare diseases. Also, Kevin Schaefer discusses how he and his physical therapist worked on him sitting unassisted during his aquatic therapy workout. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy, including its history and how it ...
9m Feb 13, 2019
9m
SMA News Today Podcast 25 - Interview With Michele Erwin
In episode 25 of the SMA News Today Podcast, Community Editor Kevin Schaefer interviews Michele Erwin, whose son Grayson has SMA. Michele is the founder and President of All Wheels Up, an organization dedicated to improving flight accommodations for wheelchair-users. In this interview, Michele talks about being a disability advocate, and how it coincides with parenting a child with SMA. The pic...
19m Feb 11, 2019
19m
Fatigue and Quality of Life in SMA Patients Not Related? & Not Letting SMA Define You as a Person
We discuss a study where perceived fatigue was not linked to physical factors regarding the quality of life in SMA patients. Also, SMA News Today’s Director of Multimedia, Michael Morale, talks about how living with SMA, has never defined him as a person. Hard work and the pursuit of success was what guided him in his life. SMA is just a byproduct of something that he has to live with. Are you...
9m Feb 11, 2019
9m
FDA's Approval for the 6MWT as Reldesemtiv Program & Setting Goals While on Physical Therapy
We discuss the FDA’s approval for the 6MWT (6 Minute Walk Test) as a primary goal of potential Reldesemtiv program for SMA patients. Also, SMA News Today’s Community Editor, Kevin Schaefer, discusses setting specific goals while taking physical therapy. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy, including its history and...
6m Feb 08, 2019
6m
SMA Types 3 and 4 Linked to Atrophy in the Neck Area & Costs of Independence When You Have SMA
Michael Morale discusses how damage that marks SMA types 3 and 4 are linked to atrophy in the neck area of spinal cords. Also, Kevin Schaefer discusses a column that he wrote regarding the cost of independence that those of us with SMA face on a day-to-day basis.
9m Feb 06, 2019
9m
SMA News & Perspectives - February 4, 2019
We discuss a newly launched website regarding patient education programs on gene and cell therapies. Also, SMA News Today’s Multimedia Director, Michael Morale, was recently asked to be the guest speaker at UT Southwestern Medical Center in Dallas Texas. Michael will be discussing the advantages of physical therapy while on treatment with Spinraza, and he will also give his suggestions as to wh...
11m Feb 04, 2019
11m
SMA News & Perspectives - February 1, 2019
We discuss how certain protein levels may track effectiveness of Spinraza. Also, Kevin Schaefer reads his article that he wrote for the SMA News Today website regarding training a new caregiver when you have SMA.
9m Feb 01, 2019
9m
SMA News & Perspectives - January 30, 2019
Michael Morale discusses how rising healthcare costs are straining the budgets in the European Union. Also, Kevin Schaefer reads from his review of the movie "The Upside", and how it portrays disability.
12m Jan 30, 2019
12m
SMA News & Perspectives - January 28, 2019
We discuss how scientists have developed a new method to evaluate nutritional status of children with SMA. Also, Michael Morale discusses the question that many individuals with SMA are trying to answer. This question involves just how far our muscles will take us in the future. We’re fortunate to have Spinraza as our first treatment option, and with the possible pending approval of ZOLGENSMA, ...
11m Jan 28, 2019
11m
SMA News & Perspectives - January 25, 2019
We discuss how ZOLGENSMA could be more cost-effective than Spinraza. Also, Kevin Schaefer discusses an article that he wrote for the SMA News Today website regarding how much each of us have changed over the past 10 years.
12m Jan 25, 2019
12m
SMA News & Perspectives - January 23, 2019
Michael Morale discusses the decision that was recently made where Poland agreed to reimburse the cost of Spinraza to treat SMA. Also, Kevin Schaefer discusses an article that he wrote for the SMA New Today forum regarding having your own space and living with SMA.
8m Jan 23, 2019
8m
SMA News Today Podcast 24 - Roundtable Discussion - The Process Of Hiring Caregivers
SMA News Today Community Editor Kevin Schaefer, and Forums & Miltimedia Director Michael Morale discuss tips on hiring a caregiver. Kevin has a caregiver that comes to his house a couple of times each day, and Michael has two live-in caregivers that are with him 24/7/365.
36m Jan 21, 2019
36m
SMA News & Perspectives - January 21, 2019
We discuss how high-resolution magnetic resonance imaging, (MRI), of the spinal cord may be a non-invasive way of monitoring SMA disease progression and responses to treatment. Also, Michael Morale discusses the importance of having SMA added to the national list of disorders that doctors use when screening newborns.
8m Jan 21, 2019
8m
SMA News & Perspectives - January 18, 2019
We discuss a new genetic approach to restore the expression of the gene that causes spinal muscular atrophy (SMA) in patients’ cells, which holds promise to effectively and permanently treat SMA. Also, Kevin Schaefer discusses the new Disability in Media forum. Kevin will be discussing the format of this new forum, which will highlight how those with disabilities are portrayed in books, movies,...
6m Jan 18, 2019
6m
SMA News & Perspectives - January 16, 2019
SMA News & Perspectives - January 16, 2019 by SMA News Today
12m Jan 16, 2019
12m
SMA News And Perspectives - January 14, 2019
SMA News And Perspectives - January 14, 2019 by SMA News Today
13m Jan 14, 2019
13m
SMA News And Perspectives - January 11, 2019
SMA News And Perspectives - January 11, 2019 by SMA News Today
9m Jan 11, 2019
9m
SMA News & Perspectives - January 9, 2019
SMA News & Perspectives - January 9, 2019 by SMA News Today
11m Jan 09, 2019
11m
SMA News & Perspectives - January 9, 2019
SMA News & Perspectives - January 9, 2019 by SMA News Today
11m Jan 09, 2019
11m
SMA News Today Podcast 23 - Interview With Tyler Dukes
SMA News Today Community Editor Kevin Schaefer interviews Tyler Dukes. Tyler has SMA Type 2, and he shares his perspectives about growing up with SMA, and the hurdles he's faced throughout his life.
54m Jan 07, 2019
54m
SMA News & Perspectives - January 7, 2019
SMA News & Perspectives - January 7, 2019 by SMA News Today
12m Jan 07, 2019
12m