Seeking help for a rare illness by going where the researchers are

Along with an article about an FDA orphan drug conference last month, The Wall Street Journal also explored how one mom is using the conference as a chance to seek treatment for her daughters by going where the researchers are and talking with them face-to-face. The purpose of the conference is to encourage researchers to pursue funding and research for orphan drugs, that is drugs for very rare diseases. The FDA has set aside substantial money for orphan drugs, but few researchers pursue it. So the FDA has hosted conferences to encourage researchers to seek the funding and help them with the application process.

But the conference isn't just attracting researchers. It has also attracted one persistent mom, who visited the conference to pursue research to find a treatment for a rare illness affecting her twin daughters. The conference helped "demystify the process" for this mom and the researchers, but also showed what can happen by actually meeting and talking to each other.