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 -
Putting the ‘Grand’ in Grandparenting
The excitement of baby news for novice grandparents rivals that of the soon-to-be parents. Giddiness pervades the pondering. Who will the baby look like? ... Read more -
Mutation Newly Linked to Adult-Onset Distal SMA in Spanish Siblings, Case Study Reports
A mutation in the KIF5A gene, found in four Spanish siblings, causes a rare form of spinal muscular atrophy (SMA) known as adult-onset distal SMA ... Read more -
Ella Learns About Purchasing Power and Budgets
The Casten household has a tight budget. I have been a teacher for 19 years, and Lindsay is the director of children’s ministries at ... Read more -
The Most Wonderful Time of the Year
For this week’s column, I’m going with something more lighthearted: existential dread. I’m kidding. My last couple of columns have been a little dreary ... Read more -
Felicia Day Taught Me to Embrace My Weird
The auditorium filled up quickly for a Monday night. Everyone was bursting with excitement. I sat in the back with my friend Jacob and ... Read more
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New Potential SMA Therapeutic Target Found in Muscle Cells, Mouse Study Suggests
Deficits in the secretion of a protein called CTRP3 by muscle cells contribute to ... -
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 ... -
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 ... -
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 ... -
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 ...
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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