To Patients

Therapeutic Approach

Restoring or Replacing Dystrophin

One of the most common types of mutations in the dystrophin gene occurs when a piece of the code in the middle of the gene is missing or deleted. By skipping additional segments of the dystrophin code called exons, the deletion can shift from an out-of-frame deletion to an in-frame deletion. Typically an in-frame deletion results in a smaller, but still functional, dystrophin protein. This shortened protein is expected to act in a similar way to normal dystrophin, and so relieve some symptoms of Duchenne and hopefully result in a more mild presentation. VILTEPSO™ is designed to skip exon 53 in the dystrophin gene.


VILTEPSO™ is an antisense oligonucleotide 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 (FDA label document). 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 additional resources regarding eligibility and access, view PPMD’s Insurance Access & Coverage Roadmap.

A Phase 3 study is actively recruiting. A Phase 2 study completed enrollment and dosing at clinical sites in North America (United States and Canada). All participants elected to enroll into an extension study. All participants continue to be dosed.


This program is sponsored by NS Pharma, Inc.

Related Studies

Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)
Extension Study of NS-065/NCNP-01

Media Library

AUG 2020

Webinar: NS Pharma Announcement of VILTEPSO™

NS Pharma joined PPMD for a webinar on August 26, 2020 to provide an update about VILTEPSO™ from Ben Yungher, PhD, NS Pharma’s Medical Science Liaison, and its associated patient support program from Randy McGonigal, NS Pharma’s Director of Market Access.
JULY 2020

NS Pharma Presents at the PPMD 2020 Virtual Annual Conference

Pre-recorded content for PPMD's 2020 Virtual Annual Conference On-Demand Library
NOV 2019

Webinar: NS Pharma Provides Viltolarsen Update

On November 20, 2019, NS Pharma joined Parent Project Muscular Dystrophy for a webinar to provide the community with an overview of its investigational drug, viltolarsen (NS-065/NCNP-01), including its mechanism of action and regulatory status. NS Pharma also provided an update on the viltolarsen clinical program, including the completed Phase 1, Phase 1/2, and Phase 2 studies, as well as an update on the viltolarsen Phase 3 trial (RACER53), including anticipated countries where study will be conducted, study design, description of the endpoints, and inclusion/exclusion criteria.
* This webinar recording contains edits to content requested by the company.
JUNE 2019

NS Pharma Presents at the PPMD 2019 Annual Conference

JUNE 2018

NS Pharma Presents at the PPMD 2018 Annual Conference

APRIL 2018

Webinar: NS Pharma's Exon 53 Skipping Program Update

NS Pharma joined PPMD for a webinar on April 18, 2018 to provide an update on NS Pharma's Exon 53 Skipping Program. Dr. Paula Clemens and Lauren Morgenroth provided the most recent updates for the trial that is ongoing in the US.
FEB 2017

Webinar: NS Pharma's Exon 53 Skipping Program - Trial Recruitment & Update

PPMD and NS Pharma hosted a webinar on February 22, 2017 to provide an update on trial recruitment for NS Pharma's Exon 53 Skipping Program.
NOV 2016

Webinar: NS Pharma's Exon 53 Skipping Program

On November 16, 2016, Parent Project Muscular Dystrophy and NS Pharma (US subsidiary of Nippon Shinyaku, Co.,Ltd) hosted a webinar to discuss NS Pharma's Exon 53 Skipping Program.

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