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 / May 14, 2025
FDA Accepts Biologics License Application for Mucopolysaccharidosis II Treatment
Our team had a wonderful time at #ASGCT2025 last week!
It was an honor to present our latest research on the promising potential of NS-089/NCNP-02 (Brogidirsen).
If you missed Dr. Aoki’s presentation, you can view it here: https://ow.ly/MbIX50VUVjA 👈
#GeneTherapy #CellTherapy https://x.com/NSPharmaInc/status/1924465920056938916/photo/1
We’re heading to Birmingham, AL, for the @JettFoundation's #FamilyWorkshop tomorrow!
Are you also attending? Make sure you stop by and say hello to our team - Patty, Yashly and Laura would love to meet you!
#DuchenneAwareness #JettFoundation #RareDiseases https://x.com/NSPharmaInc/status/1923377898716598391/photo/1
Happy 16th Birthday to our incredible #BrandAmbassador, Jordan! 🎂🎈
We hope today brings you all the joy and laughter you deserve—you’ve earned it!
Help us celebrate this incredible young man - drop your birthday wishes for Jordan below! 💙
#Birthday #ExonSkipping #Duchenne https://x.com/NSPharmaInc/status/1923030734324810226/video/1
Today is #MPSAwarenessDay!💜
MPS is a group of rare, progressive genetic disorders and we’re working hard to advance new potential #GeneTherapies for MPS I and MPS II in partnership with @REGENXBIO!
Learn more about #MPS here: https://impsn.ca/mpsday/
#MPSDAY2025 #RareDiseases https://x.com/NSPharmaInc/status/1923015523757092916/photo/1
Another big step forward for the #MPSII community!
We’re delighted to share that the FDA has accepted the Biologics License Application for RGX-121, a potential first-in-class, one-time #GeneTherapy developed by our partners at @REGENXBIO!
Learn more: https://ow.ly/btLH50VSEmF👈 https://x.com/NSPharmaInc/status/1922667899988193512/photo/1
We're in New Orleans for #ASGCT2025!
Don't miss Dr. Aoki's presentation tomorrow at 5:30pm in Poster Hall I2. They'll be sharing results from the NS-089 clinical trial, offering new insights into potential therapies for #Duchenne muscular dystrophy.
#GeneTherapy #CellTherapy https://x.com/NSPharmaInc/status/1922291545912311886/photo/1
Meet Yashly Garcia, our new Patient Engagement Lead for the South Central Region!🎉
Yashly brings years of patient experience to the team. Outside of work, she also loves to hike, try out new recipes and hang out with her pup, Rowan 🐶
Welcome to the team!
#NSPharma #NewHire https://x.com/NSPharmaInc/status/1921929194910466463/photo/1
