Updates
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From Parents’ Nightmare to Cause for Hope: The Lackey Family on Learning of SMA and Trying Zolgensma
[Editor’s Note: This is the first of a two-part series on the Lackey family of Arizona, whose daughter Stella became the first baby west ... Read more -
Higher Doses of Spinraza to Be Tested in New Phase 2/3 Trial
A new global Phase 2/3 clinical trial will test whether higher doses of Spinraza (nusinersen) provide greater efficacy to spinal muscular atrophy (SMA) ... Read more -
Ella Discovers a New Talent for Songwriting
Music helps us to express our feelings, dreams, and aspirations. Ella has been to several Young Naperville Singers rehearsals and is loving it more ... Read more -
Inhibiting Specific Cell Signaling Pathway May Help Reduce Chronic Pain in SMA, Mouse Study Shows
Inhibiting a specific signaling pathway — known as NF-κB — may help reduce chronic pain in spinal muscular atrophy (SMA), a new mouse study ... Read more -
I’m a Work in Progress, and That’s OK
A few weeks ago, someone posted a question in a Facebook group for people living with neuromuscular conditions. The question, composed of a few ... Read more -
Happiness Never Looks Like You Think It Will
As a kid, I assumed that happiness would be accompanied by a kiss in the rain, like the iconic scene between Hilary Duff and ... Read more
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MDA Awards $6.6M to Scientists Working in SMA, Other Neuromuscular Diseases
Some $6.6 million in new Muscular Dystrophy Association (MDA) multiyear grants will support promising ... -
Nice to Meet You! Greeting Someone with a Disability
Have you ever seen a person with a wheelchair rolling toward you and wondered ... -
How a Cup of Tea Helps Me to Grieve the Absence of a Caregiver
Over the winter, I drank a piping hot cup of ginger tea every night ... -
Neural Stem Cell Transplants May Delay Progression of SMARD1, Animal Study Suggests
Transplanting a subset of neural stem cells (NSCs) to the spinal canal may be ... -
Brockman Bears, Together Again
What seems a lifetime ago, my husband, Randy, and I decided to return to ... -
Life Moves Forward Despite Setbacks
The old saying, “You don’t know what you have until you lose it,” rings ...
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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