NS Pharma is working to have an important impact on the lives of patients with rare diseases. Our therapeutic areas encompass neurological diseases and inflammatory diseases.
Harnessing the power of innovative science can help bring new treatments to patients with rare diseases. NS Pharma is developing therapies through exon-skipping, cell therapy and JAK1 inhibition technology.
NS Pharma is working to have an important impact on the lives of patients with rare diseases. Our therapeutic areas encompass neurological diseases and inflammatory diseases.
Harnessing the power of innovative science can help bring new treatments to patients with rare diseases. NS Pharma is developing therapies through exon-skipping, cell therapy and JAK1 inhibition technology.
Press Release / October 14, 2025
Brogidirsen (NS-089/NCNP-02) 3.5 Year Clinical Trial Data for the Treatment of Duchenne Muscular Dystrophy Presented at 2025 World Muscle Society Congress
Press Release / October 14, 2025
Brogidirsen (NS-089/NCNP-02) 3.5 Year Clinical Trial Data for the Treatment of Duchenne Muscular Dystrophy Presented at 2025 World Muscle Society Congress
Excited to share new data at #AANEMinSanFran!
Don’t miss our poster on exon-skipping therapy persistence:
Thu 10/30 - 6:15 PM
Fri 10/31 - 2:45 PM.
Join Mara Wood & Denise Quigley to discuss insights for the #Duchenne community. See you in San Francisco! 💙
#RareDiseases https://x.com/NSPharmaInc/status/1982810230544023727/photo/1
We’re excited to attend the 10th Annual #UPINFamilyDay Conference on Oct 25 at the Loveland Living Planet Aquarium! 🐠
A day of learning, connection & fun for families impacted by neuromuscular disorders.
Register to join us: https://ow.ly/fAV150XgP4w
#RareDiseases #NSPharma https://x.com/NSPharmaInc/status/1981722931592937623/photo/1
At #NSPharma, we’re committed to advancing science & hope for #RareDiseases.
This #HunterSyndromeAwareness Week (Oct 19–25), we stand with families & advocates to raise awareness of #MPSII, a rare genetic disorder that primarily affects boys.
Learn more: https://ow.ly/1Wna50XgOW1 https://x.com/NSPharmaInc/status/1981360732408013020/photo/1
The #AANEMinSanFran Annual Meeting kicks off next week!
We’re joining global experts from Oct 30-31 to explore the latest in NM, MSK, EDX & NMUS. Don’t miss our poster (#294) where we’ll share new insights on exon-skipping for the #Duchenne community. 💙
#RareDiseases https://x.com/NSPharmaInc/status/1980998359566389570/photo/1
🌟 Welcome Melanie Towers to #NSPharma as our new Area Business Manager!
With 20+ years in sales leadership across ophthalmology, GI, hepatology & more, she brings deep expertise from Eversana, Viatris, Bausch Health & others. We’re thrilled to have you, Melanie! 💙
#NewHire https://x.com/NSPharmaInc/status/1980635721472802892/photo/1
📊 Happy #WorldStatisticsDay!
At NS Pharma, data drives every breakthrough for those living with #RareDiseases. Our analysts ensure every decision in R&D is guided by evidence; turning data into discoveries and progress for patients.
#ClinicalResearch https://x.com/NSPharmaInc/status/1980273474292662298/photo/1
October is #HunterSyndromeAwareness Month! 💜
At NS Pharma, we stand with the Hunter Syndrome community; advancing hope through our investigational gene therapy RGX-121, developed in partnership with REGENXBIO.
Learn more: https://projectalive.org/hunter-syndrome
#MPSII #RareDiseaseAwareness https://x.com/NSPharmaInc/status/1979186103799157157/photo/1
