Hot Air Balloons Adrift: Appreciating My Pulmonary Function With SMA
I gotta go climb some mountains, and I gotta do it now. That was the thought I focused on as I emerged from the pulmonary ... Read more
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Mama, It’s Amazing What SMA Patients Can Do: A Reminder of Our Adaptability
My neurologist presses his stethoscope to my chest. He’s performing a routine checkup; it’s a mere formality, really, so I can be sent home with ... Read more -
CHMP Favors EU Approval of Evrysdi as 1st Oral, At-home SMA Treatment
Evrysdi (risdiplam) has been recommended for approval in the European Union (EU) to treat spinal muscular atrophy (SMA) patients, ages 2 months and older, ... Read more -
My SMA Friends Remind Me That It’s OK to Rest
You already know that for the longest time, I didn’t want friends with SMA. I was young and desperate to be seen as “cool” (whatever ... Read more -
Evrysdi Seen to Improve Survival, Motor Development of Infants with SMA Type 1
Genentech’s Evrysdi (risdiplam) improved survival in infants with spinal muscular atrophy (SMA) type 1, and allowed them to achieve developmental milestones not usually seen ... 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 SMN1 ... Read more