When Was the First Treatment Available for SMA Patients?
The first FDA-approved treatment for Spinal Muscular Atrophy, or SMA, became available in the United States on December 23, 2016, when the FDA approved Spinraza, also known as nusinersen. Before that historic approval, people living with SMA primarily relied on supportive care such as respiratory assistance, nutritional support, physical therapy, mobility equipment, and treatment of complications. Spinraza became the first therapy specifically approved to modify the underlying disease process of SMA rather than only managing its symptoms. Cure SMA describes the 2016 approval as the first-ever FDA-approved therapy for SMA, marking a major turning point after years of research, clinical trials, advocacy, and participation from families affected by the condition.
Spinraza works by targeting the SMN2 gene, which acts as a backup to the SMN1 gene that is missing or not functioning correctly in most cases of 5q SMA. Spinraza changes the way SMN2 RNA is processed so the body can produce more complete survival motor neuron, or SMN, protein. That additional protein helps support the motor neurons responsible for controlling muscle movement. The treatment is administered directly into the cerebrospinal fluid through an intrathecal injection, and the current FDA label continues to approve Spinraza for both pediatric and adult patients with SMA. The approval showed for the first time that the biology of SMA could be directly targeted with a disease-modifying therapy, fundamentally changing expectations for patients and families.
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: