Description of Collaborative Activity: |
Neurological diseases are often chronic, slowly progressive diseases with heterogeneous populations, which present challenges when designing clinical trials of novel therapeutic or comparative effectiveness trials. The National Institute of Neurological Disorders and Stroke (NINDS) plans to further the discussion on appropriate outcome measures in such trials using Spinal Muscular Atrophy (SMA) as a sample population. The purpose of the workshop is 1) Have an interactive discussion about the current state of knowledge in the SMA field; 2) Have researchers present data on available outcome measures; 3) Have an interactive discussion on outcome measures such as patient-related outcomes/quality of life. |
