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:
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 ISIVILTEPSO 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.
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.
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.
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.