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Evrysdi Approved in Brazil for SMA, But No Indications Specified
Evrysdi (risdiplam) has been approved for the treatment of spinal muscular atrophy (SMA) in Brazil by the country’s National Health Surveillance Agency, known ... Read more -
Spinning Webs of Resilience
I’ve been grieving this month. On some days, the weather was impeccable. Morning dew twinkled on silk linens of freshly spun spiderwebs. The air ... Read more -
PAN Foundation Offers Financial Help for SMA Treatment
People living with spinal muscular atrophy (SMA) who need financial help to pay for treatment may now apply for aid through a new patient ... Read more -
After a Hiatus, Ella Sings Outdoors With Her Socially Distanced Peers
Our 10-year-old daughter, Ella, who has SMA, loves to sing. She’ll ask her mother to print out song lyrics and then sit at the ... Read more -
Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients Say
A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its ... 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