30 Million Reasons to Honor Rare Disease Day

They are diseases you’ve probably never heard of – rare conditions that afflict some 30 million Americans, many of which have no cure. Today is Rare Disease Day, a global event to raise awareness of those diseases, the patients who are suffering from them, and the urgent need for treatments and cures.

I had the opportunity to speak with one of those patients this week—Elijah Stacy, a 23-year-old man from California who is fighting to cure his disease, Duchenne muscular dystrophy. It’s a rare genetic muscle disorder that strikes children at the earliest of ages, robs them of their ability to walk, and ultimately causes their death, likely by the age of 25. You can watch our conversation here.

Despite being confined to a wheelchair since he was 11, Elijah doesn’t let his disease define him. Instead, he has made a decision to dedicate his life to an incredible mission: minimizing human suffering and propelling human prosperity. When Elijah was just 15, he founded a non-profit organization called Destroy Duchenne. At 17, he authored what went on to become a bestselling book, “A Small If: The Inspiring Story of a 17-Year-Old with a Fatal Disease-and a Mission.” He is recognized as a key opinion leader and expert on his disease, and he speaks about the promise of cutting-edge gene therapy as a means of curing rare diseases.

Elijah has also become an advocate for the Goldwater Institute’s Right to Try for Individualized Treatments (Right to Try 2.0), a law that protects patients’ right to access treatments that are tailor-made for one individual, based on their genetics. Unfortunately, patients struggle to access these treatments today because of government regulatory barriers. This week, Arkansas became the seventh state since 2022 to adopt this crucial legislation, joining Louisiana, Maryland, Mississippi, Arizona, Nevada, and North Carolina. With Elijah’s help, the Goldwater Institute will protect this right nationwide.

“Right now, I can be willing to try a new treatment, the manufacturer of the treatment can be willing to allow me to try it, and a doctor can be willing to administer it — yet Uncle Sam is not willing and stops me from receiving a lifesaving treatment,” Elijah wrote. “This is completely backwards. After all, doctors know their patients better than a far-off bureaucrat who doesn’t even know the patient exists. Why should I have to beg the federal government for permission to try to save my own life?”

On this Rare Disease Day, the Goldwater Institute thanks Elijah Stacy for his dedication and hard work in fighting for all of those suffering from rare diseases.

Mike Brownfield is the Vice President of Communications and Strategy at the Goldwater Institute.