Updates
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CSF Protein Profile May Help Predict Spinraza Responses in Late-onset SMA Patients, Study Shows
Analysis of the protein levels in the cerebrospinal fluid (CSF) of people with late-onset spinal muscular atrophy (SMA) may help to identify biomarkers that not ... Read more -
Please Stop Glorifying Being Homebound
As I write this, I’ve been home for 11 days. Actually, let me rephrase that. I haven’t left this house so much as to ... Read more -
Magnetic Rods May Be Alternative to Correct Scoliosis in Children With SMA, Study Suggests
Spine surgery using magnetically controlled growing rods (MCGR) may be an option to correct spinal deformity, or scoliosis, in young children with spinal ... Read more -
Directives that Come Straight from the Angels
Our third baby, Jeffrey, was diagnosed with spinal muscular atrophy (SMA) on a midsummer evening in 1997. The referral to a pediatric neurologist came ... Read more -
Ella Works Through Life’s Frustrations
The house is full of wheelchairs, walkers, and crutches. Soon to be added will be a cane. Ella’s mom, Lindsay, had a total left ... Read more
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Roche Starts Global Early Access Program for SMA Therapy Risdiplam in Europe
Roche has kicked off a worldwide early access program for its experimental therapy risdiplam ... -
Let 2020 Be the Year of Disability Representation
I just finished watching “The Ataxian,” a documentary from 2015 about Kyle Bryant, a ... -
Fetal Gene Therapy May Ease SMA Symptoms, Prolong Survival, Mouse Study Shows
Gene therapy administered while newborns are still in the womb may lessen spinal muscular ... -
Keeping My Goals and Adapting Them to Suit My Needs
Congratulations! You have completed another rotation around the sun. Now that the holiday celebrations ... -
NORD Webinar Features SMA Mom Discussing Zolgensma Gene Therapy
Just 27 days after Nicole Almeida gave birth to her son, Matteo, the baby ... -
ZPR1 Protein May Be New Potential Therapeutic Target for SMA, Preclinical Study Suggests
Increasing the amount of ZPR1 — a protein found in low levels in people ...
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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