Spinraza: The Treatment That Transformed SMA Forever
Before 2016, people living with SMA had supportive care for breathing, nutrition, mobility, and other complications, but there was no FDA-approved therapy capable of modifying the underlying disease process. That changed on December 23, 2016, when the U.S. Food and Drug Administration approved Spinraza for pediatric and adult patients with SMA, making it the first FDA-approved treatment for the disease. For families who had spent generations watching SMA progressively weaken muscles without a disease-modifying treatment available, the approval represented more than a medical milestone. It proved that targeted therapy for Spinal Muscular Atrophy was possible and opened an entirely new chapter of research, treatment, and hope.
Spinraza works by targeting the SMN2 gene, often described as a backup to the SMN1 gene that is missing or not functioning correctly in most cases of 5q SMA. Normally, SMN2 produces only a limited amount of complete survival motor neuron, or SMN, protein. Spinraza is an antisense oligonucleotide that changes the way SMN2 RNA is spliced, allowing the body to produce more full-length SMN protein needed to support motor neuron survival and muscle function. The treatment is delivered directly into the cerebrospinal fluid through an intrathecal injection and requires ongoing maintenance treatment. Spinraza does not restore motor neurons that have already been permanently lost, and individual results vary, but its ability to alter the expected course of SMA fundamentally changed what doctors, researchers, and families believed could be possible.
Watch Full Episode on YouTube:
Connect with Victor Guerra:
Personal Instagram:
https://www.instagram.com/Victortheinspiration/
The SMA Victor Instagram:
https://www.instagram.com/TheSMAVictor/
The SMA Victor TikTok:
https://www.tiktok.com/@TheSMAVictor
The SMA Victor Podcast Episodes:
https://www.youtube.com/playlist?list=PL7BT7VJqpaR7rASeJHidduTFEcqLSA0Ic
The SMA Victor Q&A:
https://www.youtube.com/playlist?list=PL7BT7VJqpaR7YFKqOYqs8lCeih48RO-5B
Learn More About SMA:
https://youtube.com/playlist?list=PL7BT7VJqpaR5ZI1hZ5dkVPIfjbdqvPnZE&si=WRV8gj2102jCn6mI
The SMA Victor FAQs:
https://www.youtube.com/playlist?list=PL7BT7VJqpaR4Mzd1ul1g2_XHy4Jn61oOK
The SMA Victor Music:
https://www.youtube.com/playlist?list=PL7BT7VJqpaR50uUMYbVhYsdncM-Bf1mO1
Victor's Life with SMA:
https://youtube.com/playlist?list=PLXeepRh23LFqpR6iOdsaVjgYaeRX5u_eP&si=fYKi0SN0JL1KVtcf
My book, Become the Victor: Turning Trials into Triumph, tells my personal journey and offers guidance for anyone who feels stuck, discouraged, or unsure how to move forward. It is a reminder that “your greatest tests will become your greatest testimony.” It is available online at Barnes & Noble, Amazon, iTunes, and Google Play Books.
Get My Book:
My Author Website:
https://christianfaithpublishing.com/books/?book=become-the-victor
Barnes & Noble:
https://www.barnesandnoble.com/w/become-the-victor-victor-guerra-victortheinspiration/1149224892?ean=9798901061305
Amazon:
https://www.amazon.com/gp/aw/d/B0GHB1KQNH/ref=tmm_pap_swatch_0
iTunes:
https://itunes.apple.com/us/book/isbn9798897855186
Google Play Books:
https://play.google.com/store/books/details/Victor_Guerra_Victortheinspiration_Become_the_Vict?id=YYTCEQAAQBAJ
ReaderHouse: