How can a condition that affects 30 million Americans—one in ten people—be called rare? Yet rare diseases are defined as those affecting fewer than 200,000 people. There are about 7,000 such diseases, and fewer than five percent have a treatment.

Last Wednesday, people across the U.S. gathered to call for more support, more research, and more help for families facing these conditions. Their slogan captures it well: “Alone we are rare; Together we are strong.”

At the Rare Disease Day rally in Sacramento, I met Kerry Rivas. Kerry’s son Donovan has Shprintzen-Goldberg Syndrome, a life‑threatening condition with only 70 documented cases worldwide. Getting his diagnosis took years.

Donovan has many health challenges, but the most serious affect his heart, lungs, and spinal cord. Getting him proper care is time‑consuming and expensive, forcing Kerry’s family to make major sacrifices. Even so, they work hard to give Donovan as normal a life as possible.

Although Donovan’s condition is rarer than most, everyone at Rare Disease Day shared similar stories and a strong commitment to advocacy. Their voices are being heard.

To honor the occasion, the U.S. Food and Drug Administration announced a partnership with the National Organization for Rare Disorders to hold listening sessions with patients and FDA medical reviewers.

In a news release, Peter L. Saltonstall, President and CEO of NORD, said:

“These listening sessions will provide FDA review division staff with better insight into what is important to patients in managing their diseases and improving their quality of life. It is important for FDA to understand, from the patient perspective, disease burden, management of symptoms, daily impact on quality of life, and patients’ risk tolerance. Patients and caregivers bring a pragmatic, realistic perspective about what they are willing to deal with in terms of potential risks and benefits for new therapies.”

FDA Commissioner Dr. Scott Gottlieb said his agency is committed to doing everything possible to help the rare disease community:

“Despite our successes, there are still no treatments for the vast proportion of rare diseases or conditions. FDA is committed to do what we can to stimulate the development of more products by improving the consistency and efficiency of our reviews, streamlining our processes and supporting rare disease research.”

At CIRM, we are also committed to doing all we can to help the cause. Many of the diseases we are currently funding in clinical trials are rare diseases like ALS or Lou Gehrig’s disease, SCID, spinal cord injury, and sickle cell disease.

Many pharmaceutical companies are reluctant to fund research targeting these diseases because the number of patients involved is small, making it difficult to recoup their investment or even turn a profit.

At CIRM, we don’t have to worry about those considerations. Our focus is solely on helping those in need. People like Donovan Rivas.