12/13/2025
****Recap from Nov 24th****
Well this came entirely out of left field in the SMA Community today. Itrathecal administration of gene therapy for kids older than 2yrs old was known to be in the works but over the years we hardly heard a word about trials, data, pipeline updates etc and a different treatment option was just recently denied. But then today the FDA APPROVED gene therapy treatment for SMA for kids older then 2yrs old!! The SMA community didnt even know they had submitted to the FDA! That means Grace has a shot at the gene therapy we tried so hard to get her into trials far when she was a baby before she aged out. And then was too old to receive when it was FDA approved.
No its not a cure, and we will mostly likely have to fight tooth and nail to get it for her at the rumored price tag of $2.59 million. But you bet your sweet ass we will fight for her to get it! My girl deserves EVERY opportunity possible to be stronger and to makeup for the short comings of doctors missing her diagnosis and not listening to us as parents. They are the biggest reason she never had access to gene therapy. And once neurons are lost they are gone forever, they never grow back. Access in the US isnt until some time in December but we have already got the ball rolling to figure things out with her doctors!
Please send all the good ju-ju our way that Grace will get access to gene therapy, that insurance doesnt fight us AND that we can continue her current treatment afterwards.
https://www.curesma.org/novartis-receives-fda-approval-of-itvisma-for-the-treatment-of-sma/?fbclid=IwdGRjcAOSICFleHRuA2FlbQIxMQBzcnRjBmFwcF9pZAo2NjI4NTY4Mzc5AAEeq1i2FVp0xrag7Nk8y9we7znz-ftBPKF8Dqvk322XZWvqBhr4nD9GR-_gMco_aem_-F8Htlc0PzjWxcuPEqKMSg
Novartis today announced that it has received approval from the U.S. Food and Drug Administration (FDA) for Itvisma (onasemnogene abeparvovec-brve) to treat adults and children 2 years of age and older with spinal muscular atrophy (SMA). Itvisma (onasemnogene abeparvovec-brve) is a gene t...
