Updates
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NORD’s Advice on COVID-19: You’re Stronger Than You Think
First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably have lower immunity to ... Read more -
In a Mad World, I Turn Toward Nature’s Grace
My cellphone vibrated atop my kneecap. I glanced down at the screen. An alert emerged from a local news app: A case of COVID-19 ... Read more -
FDA Issues No Fines for Data Problems in Zolgensma’s Application
The U.S. Food and Drug Administration has decided not to fine or otherwise sanction Novartis for manipulated data included in the application that led to ... Read more -
The Casten Kids Find Privacy for E-learning
School books, folders, random scratch paper, art supplies, food, drinks. All of this lay aimlessly on our kitchen table. Until recently, my children — ... Read more
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Motor Gains and Safety with Zolgensma’s IT Use Continue, STRONG Data Show
Young children given a single injection of Zolgensma into the spinal canal continue to show ... -
A Different, Better Kind of Isolation
A blue body cast covered me from my waist down. The pain in my ... -
CHMP Recommends Conditional Approval of Zolgensma for SMA in Europe
The European Medicine Agency’s Committee for Medicinal Products for Human Use (CHMP), has recommended ... -
We Can Make Something Good of This Pandemic
I cried, really cried, for the first time last week. My mom is working ... -
Things to Keep You Busy During Coronavirus Isolation
Reports about COVID-19 have been all over the news and social media. People around ... -
Zolgensma Shows Benefits and Durability Across Trials in Infants, Novartis Says
A single infusion of Zolgensma (onasemnogene abeparvovec-xioi) continues to lead to meaningful and sustained benefits ...
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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