Learning to Navigate the World in My Permobil F3
The SMA News Today Forums heard it first, but I’d like to introduce you to my brand new Permobil F3 wheelchair. Her name is ... Read more
Updates
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Immediate Treatment Advised for Newborns with 4 Copies of SMN2 in Updated Guidelines
Newborns diagnosed with spinal muscular atrophy (SMA) via newborn screening (NBS) and who carry four copies of the SMN2 gene should start treatment immediately, ... Read more -
Peering Through the Fog of Fear
I’ve been on hiatus from writing about my SMA. Truth be told, I’ve needed to break away from the constant reminder that SMA rules ... Read more -
My Introduction to Death and Beyond
My first experience with the death of someone close to me came shortly after my 16th birthday. A good friend since elementary school, Candy ... Read more -
After Loss, Life Goes on for the Castens
Life has settled down in the Casten household. We are still mourning the loss of Potter, one of our dogs. Needless to say, we ... Read more
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18 States in US Screening Newborns for SMA as Efforts Continue for More, MDA Says
Eighteen states are now screening newborns for spinal muscular atrophy (SMA), the most recent ... -
FDA Efforts to Bring Patients into Treatment Decisions Focus of NORD Webinar
The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 ... -
My Days at Ye Olde Student Newspaper
On Super Bowl Sunday in 2013, I attended my first meeting at NC State’s ... -
Targeting Defects in Cell’s ‘Skeleton’ May Restore Cellular Function in SMA, Early Study Shows
Low levels of the survival motor neuron (SMN) protein — the major root cause ... -
Enjoying an Unusual Break from the Winter Cold
A few months ago, I shared a story about a family trip to a ...
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Researchers Discover Promising New Gene Therapy Approach for Spinal Muscular Atrophy
News
A new genetic approach to restore the expression of the gene that causes spinal muscular atrophy (SMA) in patients’ cells holds promise to effectively and permanently treat the disease, researchers suggest. The study, “Seamless genetic conversion of SMN2 to ... Read more