Nikki J
Moderator
- Joined
- Mar 22, 2012
- Messages
- 17,823
- Reason
- PALS
- Diagnosis
- 04/2014
- Country
- US
- State
- MA
- City
- Boston
Mixed but hopeful results in a large and long phase 2 trial. It did not meet the primary endpoint but there was a significant survival difference between those who received drug and placebo in the pre specified slow progression group( defined by csf nfh levels. ). You will see at the end they suggest another trial might explore a more aggressive treatment to see if it could also help the fast progression group.