Rare Diseases & Orphan Drug

Biography

Jeff Leider, of Elmwood Park, New Jersey, has been on the forefront of Rare Disease Awareness for a little over five years now. His passion for advocacy began when his sons were diagnosed with MPS II/Hunters Syndrome. Since that heart wrenching day of diagnosis, he has worked tirelessly not only for his children but all children with Rare Diseases. Jeff is the President and Founder of the Let Them Be Little X2 Foundation. Mr. Leider has been on the forefront of bringing legislation to our governmental agencies. With Mr. Leider’s assistance and leadership a new law, The Let Them Be Little Act, providing testing of MPS I & MPS II in newborns, was recently signed into law by Governor Chris Christie. This is only the second state in the Union to implement this law. Mr. Leider has also been an integral part, with the support and guidance of Congressman Bill Pascrell (D-NJ9), in persuading the FDA and Shire Pharmaceuticals to open a clinical trial, allowing nine children diagnosed with Hunters Syndrome a chance to receive experimental drug to halt disease progression. Locally Mr. Leider is a Board Member for the Elmwood Park Youth Football Bombers and Officer of the Elmwood Park Little League. In November 2014, Mr. Leider was the award recipient of The Abby at the Rare Voice Awards in Washington, DC for advocacy at the State Level.

Research Interest

Rare Disease Awareness