My Greatest Dream Is to Eliminate Rare Disease Day

One of U.S. President John F. Kennedy’s dreams was to send men to the moon and have them return safely to Earth.

“We choose to go to the moon,” he told a crowd of about 40,000 people at Rice University’s football stadium in September 1962. “We choose to go to the moon in this decade and do the other things, not because they are easy, but because they are hard, because that goal will serve to organize and measure the best of our energies and skills, because that challenge is one that we are willing to accept, one we are unwilling to postpone, and one which we intend to win, and the others, too.”

Although he did not live to see the dream realized — he was assassinated in 1963, and Apollo 11 was the first manned mission to land on the moon in 1969 — President Kennedy ensured it happened because he started marshaling the people and resources. According to a 2019 article on Space.com, Kennedy believed that it was possible because NASA told him it was.

According to space policy expert John Logsdon, NASA told Kennedy, “If you commit the resources, Mr. President, we think we can do it.”

The U.S. Congress committed the resources, and NASA “did it.”

Here is my dream: that Rare Disease Day, which falls on Feb. 28, undergoes a name change one day. Right now, the day’s objective is to raise awareness about rare diseases and their impact on patients’ lives. The campaign seeks to inform both the public and political, research, and health leaders.

Having a day like this is a wonderful idea.

But it’s not enough. Statistics show that there are approximately 7,000 rare diseases that affect 25-30 million Americans. That translates to one in 10 people having a rare disease in the United States, of which 50% are children, and 30% do not live to see their fifth birthday. More than 300 million people around the world have a rare disease.

That’s too many people.

Two of these people are my wife and young adult daughter, who both have Huntington’s disease. It has been commonly described as a combination of Parkinson’s, Alzheimer’s, and ALS (Lou Gehrig’s disease).

That’s too many people too close to my heart.

And yet, like many things, rare disease research has been adversely affected since the pandemic erupted.

“Since March, medical research on diseases other than covid-19, the disease caused by the novel coronavirus, has taken a huge hit, with countless experiments abandoned and clinical trials suspended or postponed,” The Washington Post’s Laurie McGinley noted last year.

If I gave a speech to a football stadium full of medical, political, pharmaceutical, and research leaders, I would say the following: “We choose to eliminate rare diseases. We choose to eliminate rare diseases in this decade, not because it is easy to do this, but because that goal will serve to organize and measure the best of our energies and skills, because that challenge is one that we are willing to accept, one that we are unwilling to postpone, and one that we intend to win. We should all intend to eliminate Rare Disease Day and replace it with Rare Disease Day Is No Longer a Day Because Rare Diseases Are Totally Eliminated Day.”

To those reading this who have a family member with a rare disease or who are suffering from a rare disease, do not accept anything less. To those who are leaders in the political, pharmaceutical, research, or health arenas, do not accept anything less.

If man can land on the moon — if people in the 1960s accepted nothing less than that — why can’t humankind commit the necessary resources, willpower, and effort to make all rare diseases so rare that they are totally eliminated?

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Note: Huntington’s Disease News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Huntington’s Disease News or its parent company, Bionews, and are intended to spark discussion about issues pertaining to Huntington’s disease.