Exciting Progress for SMA: Four New Treatments in Development!
Apitegromab is best understood as a muscle-directed therapy. Instead of targeting the SMN1 or SMN2 genes directly, it is designed to inhibit myostatin, a protein that limits muscle growth. Its role is to potentially help improve or preserve motor function by supporting muscle strength in people with SMA, especially alongside existing SMN-targeted treatments. Scholar Rock describes apitegromab as an investigational muscle-targeted therapy that selectively targets latent myostatin, and its Phase 3 SAPPHIRE trial reported statistically significant and clinically meaningful benefit in SMA.
Salanersen is best understood as a next-generation SMN-targeted therapy. Like Spinraza, it is an antisense oligonucleotide designed to act on the backup SMN2 gene so the body can produce more functional SMN protein. Its role is to potentially extend and improve SMN-based treatment options, with Biogen describing it as an investigational therapy being studied with once-yearly dosing potential. Biogen reported that salanersen is designed to correct SMN2 pre-mRNA splicing to increase SMN protein production and received FDA Breakthrough Therapy Designation for SMA in 2026.
NMD670 is best understood as a neuromuscular transmission enhancer. Its role is different from gene replacement or SMN2 splicing therapies because it targets skeletal muscle chloride channels, specifically ClC-1, with the goal of improving how signals function between nerves and muscles. NMD Pharma describes NMD670 as a skeletal-muscle targeted ClC-1 inhibitor in clinical development for neuromuscular disorders including SMA, while clinical literature describes ClC-1 inhibition as a way to enhance neuromuscular transmission.
ARGX-119, also called adimanebart, is best understood as a neuromuscular junction stabilizer. Its role is to activate MuSK, a protein important for the structure and function of the neuromuscular junction, where nerves communicate with muscles. In SMA, this approach is being studied to see whether improving neuromuscular junction function can reduce weakness and fatigability and improve quality of life. Argenx describes adimanebart as a humanized monoclonal antibody that activates MuSK to promote neuromuscular junction maturation and stabilization, and ClinicalTrials.gov describes its SMA study as a Phase 2 proof-of-concept trial in ambulant pediatric patients.
In simple terms: salanersen focuses on SMN protein production, apitegromab focuses on muscle strength, NMD670 focuses on nerve-to-muscle signaling, and ARGX-119 focuses on stabilizing the neuromuscular junction. Together, these investigational treatments show that SMA research is expanding beyond one pathway and looking at the full disease experience: the gene, the motor neuron, the muscle, and the connection between them.
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: