SMA News & Perspectives

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...

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 st...

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.

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,...

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...

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.

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....

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 treatm...

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.

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.

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....

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.

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 h...

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.

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...

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.

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.

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, in...

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...

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...

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 ge...

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, inclu...

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 f...

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 hel...

We discuss how SMA patients in Ontario are granted wider access to Spinraza. Also, SMA News Today’s Director of Multichannel Content, Michael Morale, discusses how current treatments for SMA are giving those of us with the disease, a brighter and stronger future. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy, including its histo...