Zolgensma Has Potential to ‘Change Whole Landscape of SMA,’ Experts Say
[Editor’s note: This is the first in a series of articles on Zolgensma and treatments for SMA, the issues they raise, and possible ... Read more
Updates
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Anti-epileptic Keppra Could Be Candidate for Treatment of SMA, Study Finds
The anti-epileptic medicine Keppra (levetiracetam) may have a neuroprotective effect for spinal muscular atrophy (SMA), according to a recent study. The study, “The ... Read more -
Curiosity and Sweet Moments Build Special Bonds
As I was staring at a blank screen preparing to write this column, my little cousins Jackson and Mackenzie, who are 8 and 6 ... Read more -
Eurordis Unveils Integrated-care Initiative for Rare Disease Patients
Europe’s umbrella organization for 800 rare disease associations has developed a sweeping initiative to help the continent’s 30 million rare disease patients and their ... Read more -
Small RNA Molecule miR-23a May Have Therapeutic Role in SMA, Study Says
A small RNA molecule called miR-23a can prevent neuron degeneration and muscle wasting by acting as a protective modifier in cellular and animal ... Read more -
A Wheelchair Malfunction Reminded Me How Fragile My Reality Is
As someone living with SMA, I rely on various machines and medical equipment to function and stay alive. While I’ve never been blind to ... Read more
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From SMA to BWS: Clara’s Diagnosis Was Unexpected
We had no reason to doubt that our third baby, Jeffrey, would be healthy. ... -
Remodeling the Bathroom for Ella
Ella is growing up. She just turned 9 on June 10 and will be entering ... -
Raising Levels of Alpha-COP Protein Seen to Ease Disease Severity in Mouse Model
Elevated levels of the protein alpha-COP, involved in cellular transport and self-cleansing processes, extended survival ... -
SMA and Progression: Do What You Love While You Can
SMA comes with a lot of challenges. The progression of the disease is the ... -
My Bobblehead Days Are Behind Me
One of the ways that spinal muscular atrophy (SMA) has affected me physically is ... -
Rolling Like the Waves: SMAdventures in Adaptive Sailing
Can you recall a day that shines brightly in your mind, as you were ...
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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 ... Read more