-
-
I’m Disappointed, and That’s OK
Can I be real for a minute? Totally honest? Maybe even a little bit of a downer? I’m disappointed. 2020 was supposed to be ... Read more -
Australia Offers Subsidized Coverage of Spinraza for Pre-symptomatic Children With SMA
Australia is expanding access to Spinraza (nusinersen) to children and infants with severe spinal muscular atrophy (SMA) who have not yet developed symptoms, a period ... Read more -
Putting a Different Twist on Thankfulness
Thanksgiving is almost upon us. Meals are being planned, and turkeys are being purchased. The delicious smells and flavors of pumpkin and cinnamon will ... Read more -
Olesoxime Failed to Benefit SMA Type 2 and 3 Patients, Final Trial Data Show
Olesoxime, a small molecule once considered a potential treatment, failed to slow motor and respiratory decline in people with spinal muscular atrophy (SMA) types 2 and 3 ... Read more -
A Sigh of Gratitude After November’s Golden Yawn
They congregated on a beach in the mild November air. More than 50 of them stood there, wondering if they dare dip their feet ... Read more
Subscribe to our mailing list
Get regular updates delivered to your email box.
Thanks! Please check your email for a confirmation email.
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