You can provide immediate hope and potential treatments for ultra-rare patients. Here’s how.

Thanks to efforts of many organizations, it is now widely known that there are more than 7,000 different rare diseases that affect the lives of millions of patients and their families. But few know that there is another large and rapidly growing patient population who are even more unique and vastly more desperate that those with rare diseases: the “n-of-1” patients.

We now know that there are millions of patients that fall into a population group that may range from 1 - 30 worldwide. These genetic diseases are what we consider the ultra-rare.

We recently met with a young couple from San Diego whose daughter has a unique mutation that causes rapid aging, like Progeria. They were told there is no treatment available, and there likely will never be one. When asked what the most important thing is they would like, the mother answered, “I just want more time with her.” The father responded simply, “hope.”

As a leader in the discovery of new medicines for more than four decades, I have always believed that I work for patients. And these ultra-rare patients were asking for help. This is why I founded the n-Lorem Foundation – an organization that, like these patients, is completely unique.

n-Lorem’s mission is to provide free, lifetime supplies of experimental antisense oligonucleotide (ASO) medicines made exclusively for each patient. What we are doing has never been considered possible. Today, n-Lorem is the only organization in the world focused on this, and we have already shown that it is possible to meet the needs of these patients and families.

Rather than the one or two patients I expected to be treatable with ASOs, we will end of our first year proceeding toward treating as many as 20 of these ultra-rare patients. The number of applications will grow even more rapidly next year as we introduce n-Lorem to new groups of patients and physicians. Because the ASO technology is scalable, we can meet the demand if we have the funds.

But therein lies the challenge: we must assure that patients are not turned away because we do not have the necessary resources.

Given the expensive nature of providing personalized ASO treatments to patients for free, for life, we heavily depend on the generosity of others to help us succeed in our mission, patient by patient.

I appreciate all the support that has been so willingly provided in support our efforts to help these patients, including with our devoted partners and wonderful volunteers at Ionis. All of us on the front lines of this effort know that we could not have made the progress we have made without our many supporters, but the needs are enormous.

If you have given, we ask that you consider n-Lorem for additional support. If you are new to our quest, we ask that you please consider giving to the n-Lorem Foundation to bring hope, possibility, and treatment options to these desperate patients and families. Your tax-deductible gift will help us meet this urgent need and help save lives. I cannot imagine a better investment.

To donate, click here. For additional ways to donate or to learn more, please contact me or Tracy Johnson (tracy.johnson@nlorem.org).

On behalf of the patients and families n-Lorem serves, and my colleagues, I thank you for your consideration, support and commitment to helping those with ultra-rare diseases.