At NS Pharma we believe in the potential of new therapies, such as our exon-skipping technology for DMD, to redefine the way patients and healthcare providers fight rare diseases.

We take pride in our drive for future innovation and commitment to helping patients live healthier, happier lives.

Microscope Icon


Dedicated to improving patient outcomesMicroscope Icon

We recognize the urgent unmet need of those affected by rare diseases like DMD and are committed to fulfilling the promise of exon-skipping technology. We aim to offer healthier futures to more patients by:

  • Conducting rigorous clinical research to continue validating patient outcomes with comprehensive endpoints
  • Developing treatments that offer tangible value to those living with DMD and other rare diseases
  • Fostering a culture of scientific innovation, integrity, and continuous improvement


NS Pharma, Inc. has been developing products to change the way patients and doctors fight rare diseases. VILTEPSO® (viltolarsen), an exon-skipping therapy designed for patients with Duchenne muscular dystrophy (DMD), was granted accelerated approval by the FDA. There is an ongoing study to confirm the clinical benefit of VILTEPSO.


VILTEPSO is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with VILTEPSO. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

For more information about VILTEPSO, see full Prescribing Information.

Important Safety Information


In clinical studies, no patients experienced kidney toxicity during treatment with VILTEPSO. However, kidney toxicity from drugs like VILTEPSO may be possible. Your doctor may monitor the health of your kidneys before starting and during treatment with VILTEPSO.

Common side effects include upper respiratory tract infection, injection site reaction, cough, and fever.

For more information about VILTEPSO, see full Prescribing Information.


Building upon a 100-year legacyPeople Icon

Our corporate philosophy is derived from the extensive history and established relationships of our parent company, Nippon Shinyaku. We continue to build upon the legacy of Nippon Shinyaku to make a lasting, positive impact on all stakeholders.


Delivering life-changing medicines with the quality and safety that patients and caregivers can rely on.


Earning and maintaining Shinrai—the Japanese word for “trust”—in the rare disease community by acting with integrity in everything we do.


Cultivating the passion and solidarity of our employees, who work together, every day, to advance our mission.


NS Pharma is fully committed to ensuring that all of our activities comply with all applicable laws and regulations. Learn more

The following principles are at the heart of our company’s culture:

Meet challenges

We will always take a positive approach in pursuing our goals, with a sense of responsibility rooted in our ethical obligations.

Swift action

We will always move quickly in order to seize opportunities to serve the people and communities we stand with.

Spirit of innovation

We will always strive for Dokujisei—the Japanese word for “originality”—as we explore unique and creative solutions in health care.

Keep on smiling

We will always approach every situation—especially the difficult ones—with a smile on our face.

Lightbulb Icon


Raising the bar in the fight against DMD and other rare diseasesLightbulb Icon

At NS Pharma we delve deeper to bring products to market that add demonstrable value to the community. Our dedicated team of pioneering scientists is advancing the frontiers of exon-skipping technology.

In our relentless pursuit of groundbreaking exon-skipping therapies, NS Pharma has developed a uniquely optimized approach to drug discovery. This proprietary process identifies the highest possible exon-skipping efficiency through a systematic, three-step screening process.


Together, we can support the needs of the communityHandshake Icon

We are dedicated to being a committed partner to the families and communities impacted by rare diseases, including DMD. We are working with leading rare disease advocacy organizations to help facilitate access to treatments such as exon-skipping therapy and patient support.

NS Pharma is proudly partnering with the following organizations: