The Hospital for Sick Children (SickKids) in Toronto is currently recruiting boys with Duchenne muscular dystrophy aged 4-7 with mutations amenable to exon 53 skipping for the NS Pharma, Inc. RACER53 clinical trial investigating the new drug, viltolarsen.
Click here to learn more and find out if you might qualify for this study: racer53dmdstudy.com.
RACER53 Study Overview
RACER53 stands for “Reassemble And Correct Exon Reading 53” and is a phase 3 randomized, double-blind, placebo-controlled, multicenter study to assess the efficacy and safety of viltolarsen in ambulant boys with Duchenne muscular dystrophy (DMD).
Approximately 74 individuals will participate in RACER53 across 41 clinical sites located in approximately 16 countries, predominantly in the European Union (EU), Asia (Japan, Australia, Korea), North America (the United States [US] and Canada), and potentially South America.
All patients that complete the 1 year blinded study will be eligible to enter a 2 year-long open-label extension study where all patients will receive viltolarsen IV weekly.
What is viltolarsen?
Viltolarsen is an “investigational” drug, which means it has been approved for research purposes only, and can only be prescribed by a study doctor.
Viltolarsen is known as an “exon skipping agent.” It is designed to help the cells “skip” over missing or incorrect sections of genetic code, so the body still can create a functional dystrophin protein.
Viltolarsen is given as a weekly, 1-hour, intravenous (IV) infusion into the blood.
If you have any questions, please reach out to:
Director of Research and Advocacy