Building healthier futures powered by thoughtful science

We are a highly focused, research-driven biopharmaceutical company working in rare diseases. We're exploring the potential of cutting-edge science as a subsidiary of Nippon Shinyaku Co., Ltd. In Japan.

Advancing rare disease research

Inspired by our own families, as well as the individuals we aim to provide with treatment, NS Pharma is conducting advanced research in rare diseases, including exon-skipping technology, to offer new, effective treatment options for Duchenne. We’re also developing JAK1 inhibition for the potential treatment of eosinophilic granulomatosis with polyangiitis (EGPA).

Therapeutic areas

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.

Scientific approaches

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.

Therapeutic areas

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.

Scientific approaches

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.

From our blog

Press Release / July 1, 2025

NS Pharma Announces Strategic Alliance with Boston Children’s Hospital to Develop New Therapies for Rare Diseases

Press Release / May 14, 2025

FDA Accepts Biologics License Application for Mucopolysaccharidosis II Treatment

Press Release / April 18, 2025

FDA Grants Orphan Drug Designation to NS-229 for the Treatment of Eosinophilic Granulomatosis with Polyangiitis

Press Release / July 1, 2025

NS Pharma Announces Strategic Alliance with Boston Children’s Hospital to Develop New Therapies for Rare Diseases

Press Release / May 14, 2025

FDA Accepts Biologics License Application for Mucopolysaccharidosis II Treatment

Press Release / April 18, 2025

FDA Grants Orphan Drug Designation to NS-229 for the Treatment of Eosinophilic Granulomatosis with Polyangiitis

Social media

Did you know that September is #NewbornScreeningAwarenessMonth?

Right now, there is an opportunity to add #Duchenne to the national #NewBornScreening panel and your voice can help make this change a reality.

View the petition: https://ow.ly/cSJ650WP6s6

#DMD #EarlyDetection https://x.com/NSPharmaInc/status/1962878661335359729/photo/1

We're on our way to Kyoto for #ICIEM2025!🎉

As proud sponsors, we can’t wait to get involved, learn from the latest science, and keep moving forward together for patients and families living with inborn #MetabolicDisorders.

If you’re attending, let’s connect!💙

#RareDiseases https://x.com/NSPharmaInc/status/1962518696607309918/photo/1

Did you know #Duchenne is a rare genetic disorder that almost always affects boys?🔎

As we approach #WorldDuchenneAwarenessDay, we’re sharing some important facts about #DMD to help raise awareness. Check them out below!

Learn more: https://ow.ly/O9f450WNKlH

#RareDiseases https://x.com/NSPharmaInc/status/1961125220594466859/video/1

Please join us in welcoming Buddy Britton to team as our newest Key Account Manager!

His deep expertise in cardiology, oncology, neurology and #RareDiseases will be a tremendous asset to our team!

Outside of work, you'll often find him boating, fishing or golfing.

#GrowingTeam https://x.com/NSPharmaInc/status/1959979409911230622/photo/1

We’re honored to sponsor this weekend’s CureDuchenne Cares Workshop in Orange County!

Join us for a day of expert insights, hands-on learning, and meaningful connection!

#CureDuchenneCares #DuchenneAwareness #DuchenneCommunity #RareDisease https://x.com/NSPharmaInc/status/1958892262189842742/photo/1

You’ve probably never thought much about #Dystrophin - but for your muscles, it’s everything.

Here's 5 key facts about dystrophin and why this tiny protein plays a massive role in neuromuscular health!

Learn more: https://ow.ly/Mfax50WHwrW

#RareDiseases https://x.com/NSPharmaInc/status/1958167331739148332/video/1

Meet Michele McFarlane!

Michele joins our Commercial Sales team as the newest Key Account Manager with more than 20 years experience in #RareDisease advocacy!

Outside of work, she enjoys hot yoga and live country music with her husband Don.

#Welcome, Michelle!

#NewHire https://x.com/NSPharmaInc/status/1957443039355433177/photo/1