Neena Nizar Secures NIH Trial for 1-Patient Jansen's Disease Study
Updated
Updated · The New York Times · Aug 17
Neena Nizar Secures NIH Trial for 1-Patient Jansen's Disease Study
2 articles · Updated · The New York Times · Aug 17
Summary
Ten weeks at the National Institutes of Health made Neena Nizar the sole participant in an experimental drug trial for Jansen’s disease, a genetic disorder known in only about 30 people worldwide.
Nizar is not expected to reverse her own severe bone damage, but researchers hope the drug could work well enough for children to enter the study next and avoid the disease’s worst effects.
Her case underscores how the trial existed largely because of her yearslong push for attention and funding, highlighting the unusual burden patients can face when a disease is too rare to attract a viable market.
An estimated 6,000 to 10,000 rare diseases remain in that bind, with single-drug development often costing millions of dollars even before advocates find a manufacturer and clinicians to run trials.