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What Home Schooling Was Like for Me
I was a sophomore in high school when my parents and I agreed to home-school me during the winter months. It was a spur-of-the-moment ... Read more -
Novartis to Open, at FDA Request, New Trial of Zolgensma for Older Patients
Novartis will open a new clinical trial investigating Zolgensma, given via an injection directly into the spinal canal, after the U.S. Food and Drug ... Read more -
Spinraza Leads to Motor Gains Over Time in SMA Type 3 Adults, Real-Life Study Finds
About one year of treatment with Spinraza (nusinersen) gradually and significantly improves motor function in adults with spinal muscular atrophy (SMA) type ... Read more -
Ella Creates Special Bonds With Her Pets
My 10-year-old daughter with SMA, Ella, loves animals, so much so that she has a computer game simulating the adoption, trading, and buying of ... Read more
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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