|
Heather R. Adams |
Outcome Measures and Infrastructure for Phase III Studies in Batten Disease (JNCL) |
|
Jonathan W. Mink |
Natural History of JNCL and other NCLs |
|
Robert C. Griggs |
The Experimental Therapeutics of Rare Diseases |
|
Heather R. Adams |
Development and Validation of Clinical Trial Endpoints |
|
Lisa de Blieck |
Patient Registries in Rare Diseases |
|
Erika F. Augustine |
Widely Used Clinical Endpoints for Neurologic Disease – Applications for JNCL |
|
Ellen Bletsoe |
Phase III Clinical Trials in JNCL: Treatment Outcomes & What is Most Meaningful to Families |
|
Lori Sikorra |
Take Your Time |
|
Michael P. McDermott |
Clinical Trials in Rare Diseases: Challenges in Design, Analysis, and Interpretation |
|
David Pearce |
Preclinical Endpoints in CLN3 |
|
John F. Staropoli |
The MGH NCL Biorepository: Development of Preclinical Screening Platforms and Biomarkers |
|
Angela Schulz |
DEM-CHILD (for information about DEM-CHILD, please see the website for the project) |
|
Angela Schulz |
Review of Hamburg NCL Rating Scale |
|
Jonathan W. Mink |
Review of UBDRS in JNCL: Reliability, Validity, and Endpoints |