Yesterday, the drug BPN14770, which is in development for possible treatment of Fragile X syndrome, was granted orphan drug status by the FDA. As the patient advocacy organization for Fragile X, the NFXF will be assisting in recruiting participants for the trial.

Orphan drugs are drugs being developed for disorders/diseases that affect less than 200,000 individuals in the U.S. Research and development is sparked by vital laws that provide tax credits and other incentives to compensate for the small markets. NFXF Advocates continue to work to promote these policies in Congress, including retaining an Orphan Drug Tax Credit during the tax reform legislation last year and advocating for the OPEN Act this year.

The path to effective treatments and a cure is long and requires efforts from all angles – research, federal and private funding, public policy and legislative advocacy, and patients to participate in trials. Together, we can Stand Up to Fragile X.

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