FDA Approves Risdiplam, as Evrysdi, 1st Oral Treatment for All SMA Types
The U.S. Food and Drug Administration (FDA) has approved Evrysdi — formerly known as risdiplam — as the first oral and at-home treatment for ... Read more
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SMA Foundation’s ‘Little Toolbox’ Helped Pave Way to Therapies
[Editor’s Note: This is part of a series of articles into the discovery and development of Evrysdi, SMA’s newly approved disease-modifying therapy and its ... Read more -
31 Days of SMA: Amber Bosselman
View this post on Instagram Day 11 of 31 Days SMA Topic: Embracing Life with SMA This is Amber Bosselman (@ambercarleen ) story: I ... Read more -
New Streaming Channel Showcases Rare Disease Films
When the COVID-19 pandemic forced the postponement of a rare disease film festival originally slated for May, its organizers set out to find a ... Read more -
31 Days of SMA: Heather Kerstetter
View this post on Instagram Day 10 of 31 Days SMA Topic: Being a Social Worker with SMA This is Heather Kerstetter (@letsdothisheather ) ... Read more -
Facing My Fear of Surgery Through British TV
I was a bit of an Anglophile in high school. I was obsessed with anything produced by the BBC — “Doctor Who,” “Sherlock,” “Merlin,” ... 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