Amit Rakhit, MD, MBA: Measuring Improvement in Fragile X Syndrome, Other Rare Conditions

“Part of the research that we’ve done here is revealing that interviewing individual family members who take care of their loved one with Fragile X syndrome. What’s important for them?”

There are a number of rare genetic conditions faced by neurologists, clinicians, and health care practitioners which are severely limited in available treatments. Making this even more difficult is that they lack valuable and effective measurements to use in clinical trials.

This is the case in Fragile X syndrome, also known as Martin-Bell syndrome. To help address this, Ovid Therapeutics assessed what been done in previous trials of Fragile X syndrome to establish a Visual Analog Scale, incorporating behavior, cognition, speech and communication, sensory perception, emotions, social, activities of daily living (ADL), and notably, caregiver impacts.

To find out more about this scale and what prompted the incorporation of patient and caregiver centered facets into it, NeurologyLive^® spoke with Amit Rakhit, MD, MBA, chief medical officer, and head, Research & Development, Ovid Therapeutics, at the 2019 American Academy of Neurology (AAN) Annual Meeting in Philadelphia, Pennsylvania.

Rahkit additionally explored how this methodology could be adopted to develop similar scales in other conditions which lack an effective measurement tool.

For more coverage of AAN 2019, click here.

REFERENCE

Lee A, Cordeiro L, Marshall C, Wilson E, Visootsak J. Development of a Conceptual Model to Inform a Clinical Outcome Assessment Strategy in Adolescents and Young Adults with Fragile X Syndrome. Presented at: 2019 American Academy of Neurology Annual Meeting. May 4-10, 2019; Philadelphia, PA. Poster 067.