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

Shout-out to @TeamJosephInfo for the thoughtful gift packages sent ahead of last week’s #CommunityCampfire!

Our team always has a great time connecting with this vibrant community, and we are proud to continue driving the conversation about what’s possible.

#Duchenne #DMD https://x.com/NSPharmaInc/status/1948020284902305892/photo/1

2

Say hello to Lisa Weaver, our new Director of Patient Access!

Lisa brings over two decades of experience in pharma account management, reimbursement strategy and patient access.

We’re thrilled to have her join the #PatientServices team.

#Welcome aboard, Lisa!

#NSPLife https://x.com/NSPharmaInc/status/1947658363904352323/photo/1

A warm welcome to our new Director of Pharmacovigilance on the #MedicalAffairs team, Nora 👋

Norma brings 15+ years of experience in clinical safety, regulatory reporting and adverse event management. She loves spending time with her family, cooking and exploring new places! https://x.com/NSPharmaInc/status/1946208751012184413/photo/1

Starting tomorrow, Team Joseph kicks off the second annual What Now Conference!

It's a FREE and 100% virtual event created by and for older teens and adults living with Duchenne.

Register now to get involved: https://ow.ly/3NGC50Wp4Uw

#Duchenne #RareDiseases #VirtualConference https://x.com/NSPharmaInc/status/1945121807947747579/photo/1

Recently, NSP team members Patti Santiago and Latoya Martin had the privilege of spending time with one of the incredible campers at #CampPromise!

A huge thank you to the @JettFoundation for building a space filled with joy, laughter and inclusion. 💙

#Duchenne #RareDisease https://x.com/NSPharmaInc/status/1945121171072278859/photo/1

Are there ways to increase levels of dystrophin?🧬

Yes - #ExonSkipping and #GeneTherapy!

Both approaches aim to improve muscle function and slow progression.

Learn more about these options and how they work by visiting: https://ow.ly/wWxc50Wiiyc

#DuchenneAwareness https://x.com/NSPharmaInc/status/1943671824597778906/photo/1

Say hello to Jodie Danko, our new Senior Director of #PatientServices!🌟

With 20+ years of experience in the industry, Jodie brings deep expertise and a passion for improving the patient journey.

Outside of work, she enjoys boating and water sports!

#RareDiseaseCare https://x.com/NSPharmaInc/status/1942238855966409099/photo/1