Rare Diseases

Ella Murray is a happy, inquisitive elementary school student. However, because she was born without collagen factor VII, she suffers from a disease called epidermolysis bullosa (EB). This causes her to develop blisters all over her body, and she has to endure 3 hours of bandage changes multiple times a week. There are currently no therapies for EB, in part due to low prevalence of the disease and low enrollment in clinical trials. Joe Murray, Ella’s dad, told her story and called on researchers to inform communities about existing clinical trials during the Coalition for Clinical Trials Awareness panel discussion on child inclusion in clinical trials held in Washington, D.C. on May 1. This...

This blog post originally appeared August 3, 2016 on the Sick Chicks and The Mighty . "Congress is working together on a nonpartisan issue that will have a profound effect on the lives of all Americans. H.R. 6, the 21st Century Cures Act, will bring our health care innovation infrastructure into the 21st Century, delivering hope for patients and loved ones and providing necessary resources to researchers to continue their efforts to uncover the next generation of cures and treatments." - Mission Statement , House Committee of Energy Commerce, 21st Century Cures Is it just me or do you get chills reading that paragraph? Finding advocacy allowed me take control of an uncontrollable situation...

Guest blog post by the National Organization for Rare Disorders. February 28 is Rare Disease Day ’€“ a time to show support for the 30 million Americans, and millions more around the world, living with rare diseases. Only a few hundred of the nearly 7,000 rare diseases have approved therapies. Many are not being studied at all by medical researchers. Often, patients ’€“ or parents of patients ’€“ feel that nothing will be done unless they raise the money, recruit the researchers and fuel the search for their own lifesaving treatment. They find themselves in a race against time, since rare diseases tend to be serious and many are life-threatening. To promote awareness of these and other rare...

Dear Research Advocate: Our elected representatives know they must make hard tax and entitlement reform decisions, and, for the sake of the nation, ensure those decisions foster economic growth and societal progress. Part of that equation is federal funding for medical research sufficient to capitalize on unprecedented scientific opportunity and tackle urgent threats like Alzheimer’€™s Disease. As I’€™ve highlighted before, a majority of Americans say they are willing to pay additional taxes ’€” $1 more per week (which amounts to approximately $4.4 billion annually) ’€” if they knew those dollars were funding medical research. The public is on our side with their wallets as well as their...