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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The Struggle of Getting Medical Equipment
My life is full of medical appointments, and they all tend to be fairly inconvenient. Necessary, but inconvenient. I can’t attend them on my ... Read more -
I’m Learning to Recognize My Own Limitations
Since I started receiving my Spinraza (nusinersen) treatments in November 2017, my energy level has skyrocketed. Often, I don’t even realize just ... Read more -
Days That End in ‘Why’: A Letter to My Parents
Dear Mom and Dad, In the wake of FDA approval of Zolgensma, jubilant feelings soar like a kite, beyond the boundaries of wonderful to ... Read more -
Finding Serenity in the Eye of the Storm
If you’ve been keeping up with my column, you’ll know that my parents and I are essentially living in a no-fly zone (only our ... Read more -
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
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Curiosity and Sweet Moments Build Special Bonds
As I was staring at a blank screen preparing to write this column, my ... -
Eurordis Unveils Integrated-care Initiative for Rare Disease Patients
Europe’s umbrella organization for 800 rare disease associations has developed a sweeping initiative to ... -
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 ... -
A Wheelchair Malfunction Reminded Me How Fragile My Reality Is
As someone living with SMA, I rely on various machines and medical equipment to ... -
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 ...
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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