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Productivity and Survival Are 2 Sides of the Trauma Response Coin
I keep hearing about “making the most of quarantine.” Learn a new skill! Finish that book you’ve been reading for months! Plant a garden! ... Read more -
Lack of Muscle Cell Signaling Seen to Kill Motor Neurons in SMA-LED2
Problems with the secretion of survival signals from muscle cells seems to be the dominant driver of motor neuron loss in spinal muscular atrophy ... Read more -
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 -
First Patient Treated in Phase 2/3 Trial Assessing Higher Dose of Spinraza
The first patient has been dosed in the Phase 2/3 DEVOTE trial assessing the safety, tolerability, and efficacy of a higher dose of ... 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
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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 ... -
The Casten Kids Find Privacy for E-learning
School books, folders, random scratch paper, art supplies, food, drinks. All of this lay ... -
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 ...
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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