Interventions of Interest:
- atidarsagene autotemcel (“arsa-cel”, Orchard Therapeutics)
For questions please contact Kelsey Gosselin, Program Manager, at kgosselin@icer.org.
View the Key Stakeholder List.
Metachromatic Leukodystrophy
An assessment of the gene therapy, atidarsagene autotemcel (“arsa-cel”)
Public Meeting
September 2023
Assessment Status
Open
Upcoming Milestone
Revised Scoping DocumentView
Main Review
September 29, 2023
CTAF
The CTAF’s 2023 public meeting on metachromatic leukodystrophy.
Public Comment Period
See Full Timeline
03/09/2023 - 03/30/2023
Public Comments on Draft Scoping Document
Related Assessments & Policy Papers.
Duchenne Muscular Dystrophy
Jul 2019Spinal Muscular Atrophy
Mar 2019
03/09/2023 - 03/30/2023
Public Comments on Draft Scoping Document
04/06/2023
Revised Scoping Document
04/13/2023
Research Protocol
Please find the Research Protocol here:
06/01/2023
Model Analysis Plan
Please find the Model Analysis Plan here:
07/26/2023 - 08/22/2023
Public Comments on Draft Evidence Report
09/14/2023
Evidence Report
09/29/2023
Evidence Presentation
10/30/2023
Public Comments
OpenView
Closed