Our focus

At NS Pharma we believe in the potential of new therapies, such as our exon-skipping technology for Duchenne muscular dystrophy, 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.

OUR COMMITMENT

Dedicated to improving
patient outcomes

We recognize the urgent unmet need of those affected by rare diseases like Duchenne 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
  • Developing treatments that offer tangible value to those living with Duchenne and other rare diseases
  • Fostering a culture of scientific innovation, integrity, and continuous improvement

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.

Our products

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. Preliminary results from a Phase 3 confirmatory study of VILTEPSO have been received and are undergoing analysis and discussion with the FDA.

View indication and ISI

Indication

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.

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.

BOLD INNOVATION

Raising the bar in the fight against Duchenne and other rare diseases

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 screening process.

COMMUNITY PARTNERSHIP

Together, we can support the needs of the community

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