By Hilary Rosselot

Advocates, your hard work paid off!

Year after year we advocate with our Members of Congress to keep Fragile X as an eligible condition for funding from the Department of Defense (DOD) Congressionally Directed Medical Research Program (CDMRP) Peer Reviewed Medical Research Program (PRMRP). We have to have at least one champion suggest Fragile X stay on the list in order to be eligible for funding. For several years now, NFXF Advocates have been able to secure a champion and keep Fragile X on the list.

That work as paid off; PureTech Health has been awarded a grant of up to $11.4 million from the DOD for their trial of LYT-300, oral formulation of allopregnanolone, in people with Fragile X-associated tremor/ataxia syndrome (FXTAS).  PureTech is working alongside the team at the UC Davis MIND Institute with Dr. Randi Hagerman as a co-co-principal investigator. The first year of the DOD grant is focused on identifying biomarkers. If all goes well, the research will move forward.

FXTAS has no targeted treatments and families are anxiously awaiting relief. This grant is an incredible step in the right direction.

Congratulations to the PureTech and UC Davis Mind Institute teams on this funding! And thank you, Advocates, for your tireless efforts to improve the lives of those living with Fragile X.

about
Author Hilary Rosselot

Hilary Rosselot
Hilary joined the NFXF team in 2019. Prior to joining the NFXF team, she worked at the Cincinnati Fragile X Research and Treatment Center for over five years. She has experience as a clinical research coordinator across many types of clinical trials and served as the clinical research manager for the Cincinnati program. She earned a bachelor’s degree in psychology, a master’s, and is a SOCRA certified clinical research professional (CCRP). She enjoys time with family and friends, a great book, a strong cup of coffee and, of course, a good laugh!

learn more

Research Funding Opportunity from CDMRP/PRMRP for Fiscal Year 23 | NFXF (fragilex.org)
Explanation of  FY23 CDRMP/PRMRP opportunities

Study: Mechanisms and biomarkers of disease progression in Fragile X-associated tremor/ataxia syndrome (FXTAS)

The University of Kansas BRAIN Lab is conducting a research study to learn about behavioral and brain differences associated with the Fragile X premutation. Males and females ages 50-80 living with the Fragile X premutation, with or without FXTAS, may be eligible to participate. The study includes remote & in-person visits at the University of Kansas.