Shots:

• The first patient has been dosed in P-III CIFFREO study assessing PF-06939926 vs PBO in 99 ambulatory male patients aged 4-7yrs. with DMD across 55 sites in 15 countries.
• The first patient was dosed at a site in Barcelona- Spain on Dec 29- 2020. The 1EPs of the study is the change in NSAA @1yr- while patients will be followed in the CIFFREO study for 5yrs. after treatment with PF-06939926
• PF-06939926 is an investigational rAAV9 capsid carrying a shortened version of the human dystrophin gene (mini-dystrophin) under the control of a human muscle-specific promotor and has received the US FDA’s FT designation in Oct’2020 and ODD & RPD in May’2017

 ­ Ref: Businesswire | Image: Wall Street Journal