Ongoing Clinical Trials
**** Copied verbatim from AveXis press release (Link in HELPFUL LINKS page) ****
Estimated Enrollment: 44 participants
Actual Study Start Date: September 29, 2017
Estimated Primary Completion Date: March 31, 2020
Estimated Study Completion Date: March 31, 2020
Ages Eligible for Study: up to 180 Days (Child)
Estimated Enrollment: 27 participants
Actual Study Start Date: December 14, 2017
Estimated Primary Completion Date: August 30, 2019
Estimated Study Completion Date: August 30, 2019
Ages Eligible for Study: up to 60 Months (Child)
Planned Trials In SMA ( I will update with trial identifiers when each trial starts )
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In this week’s video, I discuss the REALITY of what Spinraza and Evrysdi ACTUALLY do for those of us with SMA.
I discuss my thoughts regarding these treatments, and I also discussed how hopeful we should all be with regards to Scholar Rock’s new treatment known as apitegromab.
This potential treatment will be the first of its kind because it will be a muscle targeted therapy, that will be used in combination with Spinraza, and other possible treatments.
Be sure to read Emily's latest article: