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 -
My Family Is Thankful for the Nurses Who Care for Me at Home
In January 2011, I had a period of illness that sparked an emergency tracheostomy surgery. It also marked the time that home nursing care ... Read more -
Faced with the Unexpected, My Family Had to Break Our ‘Sick Rules’
It has been a bizarre week in the Silva household. Come to think of it, “stressful” may be a better adjective to describe the ... Read more -
Ella and Henry Make Me Proud at Their Parent-teacher Conferences
The school was bustling as children and adults exchanged greetings while rushing about. Parent-teacher conferences were underway. I arrived at the school with Ella ... Read more -
SMN Protein Study in Fetuses and Children Emphasizes Importance of Early SMA Treatment, Newborn Screening
Levels of the survival motor neuron (SMN) protein — which are significantly low in spinal muscular atrophy (SMA) patients — naturally rise during gestation and ... Read more
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Spinraza Improves or Stabilizes Motor, Lung Function in SMA Type 3 Adults, German Study Suggests
Spinraza (nusinersen) improves or stabilizes motor and lung functions in adults with spinal muscular atrophy (SMA) type ... -
The Interactions That Stay with Me
A few weeks ago, my mom and I went to Junk Bonanza, a vintage ... -
On Fulfilling My Role as Maid of Honor
“Houston, we’re not in kindergarten anymore,” I said as we looked at our reflections ... -
Zolgensma Produces Promising Results in Young Children with Type 2 SMA, Early Trial Data Show
A single injection of Zolgensma into the spinal canal (intrathecal) can provide a clinically ...
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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