Skip to main content
Utility Menu
Patients
Investors
Newsroom
Contact Us
Regional Navigation
Global Sites
Switzerland
Deutsch
English
Français
Italiano
UK
English
United States
English
Toggle Menu Button
Utility Menu
Patients
Investors
Newsroom
Contact Us
Regional Navigation
Global Sites
Switzerland
Deutsch
English
Français
Italiano
UK
English
United States
English
Social Links
Twitter
LinkedIn
Instagram
Facebook
Main navigation
Our Disease Areas
Duchenne Muscular Dystrophy
Limb-girdle Muscular Dystrophy
Charcot-Marie-Tooth Disease
Our Science
Gene Therapy Engine
RNA Platform
Gene Editing
Manufacturing
Strategic Partnerships
Investigator-Initiated Studies
Our Products & Pipeline
Products
Pipeline
Clinical Trials
Treatment Access
About Us
Leadership
Patient Affairs
Corporate Responsibility
Grants & Giving
Global Locations
Contact Us
Join Us
Career Opportunities
Toggle Menu Button
Pipeline Bar Color
#661b62
Other Exon Targets²
Read more
about Other Exon Targets²
CRISPR/CAS9 - Harvard University
Read more
about CRISPR/CAS9 - Harvard University
CRISPR/CAS9 - Harvard University
Read more
about CRISPR/CAS9 - Harvard University
VYONDYS 53 (golodirsen)²
Read more
about VYONDYS 53 (golodirsen)²
AMONDYS 45 (casimersen)²
Read more
about AMONDYS 45 (casimersen)²
SRP-5051 (vesleteplirsen)
Read more
about SRP-5051 (vesleteplirsen)
CRISPR/CAS9 - Duke University
Read more
about CRISPR/CAS9 - Duke University
EXONDYS 51 (eteplirsen)²
Read more
about EXONDYS 51 (eteplirsen)²
Subscribe to Duchenne
