Reimagining the Future of Rare

Forty years ago, the Orphan Drug Act was enacted, becoming the world’s first law to encourage developing treatments for rare diseases. Shortly afterward, the coalition of patient advocates who had championed this cause established NORD to provide a unified voice for all Americans with rare diseases. The grassroots movement that led to the ODA and NORD was a turning point and a new beginning for those affected by rare diseases. They were no longer alone. Their voices were now being heard. And the movement that began 40 years ago continues to grow today.

40 Years of Impact Driven by You

Before the Orphan Drug Act became law, fewer than 40 drugs were developed to treat rare diseases. Today, more than 600 treatments have been  approved by the Federal Drug Administration (FDA) for rare diseases. The rare community has also made great strides in many other areas, including education, public policy, and patient/family services. As the community celebrates 40 years of progress, these milestones reflect the dedication and passion of the patients, caregivers, researchers, clinicians, and government and industry partners who have made this journey together.   

Scroll to explore NORD’s 40-year history and major milestones that shaped the rare disease community:

Ways to Get Involved
As we boldly reimagine the future, we invite you to join us in shaping what is possible for yourself, your loved ones, and the entire community affected by rare disease.

Join Us

Volunteer with NORD or attend an in-person or virtual event this year. From raising awareness to advocating for state and federal policies, there are options for everyone – including organizations seeking to become members.

Get Involved

Share Your Story

Join the individuals and families in the NORD community who have contributed their personal accounts of living with rare diseases. Help us shine a light on rare diseases by voicing your journey for others. Alone we are rare. Together we are strong!

Share Story

Ways to Give

You can support the rare disease community by starting a fundraiser to support NORD or by making your own gift today. All support helps improve the quality of life and access to education and care for our rare disease community members. 

Donate Today

Reimagining Rare

Hover and click to read shared community stories.
Click here to tell us how you are reimagining rare.

Jeron Hill

More access to treatment and resources for patients who are new to the community and...

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Sam Curtis

I’d rather focus on the rare disease community than focus on my own rare condition...

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Stephanie Levin

I was diagnosed with Pompe Disease in February 2021. I’ve probably been symptomatic for about...

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Megan Davis

For physicians to be educated on scleroderma. We are not lazy, crazy, seeking attention! We...

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Sarah Foye

I envision a future where people with rare muscle disorders like titinopathy (caused by disease-causing...

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Cristina Rosa

I am an advocate for legislation pertaining to rare diseases and the parent of Juju,...

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Lisa Zaret

I would like more attention called to West Nile virus and the rarest manifestation, meningoencephalitis.

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Kala McWain

Better mental and psychosocial support for caregivers.

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Julie Garza

Mask mandates in all medical settings would make an enormous difference to immunosuppressed patients.

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Karen Quandt

The price of rare disease drugs is extremely high. How do we fix this problem?

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Hopefully government and private sources will soon have designated funds to help cure Rare Diseases.

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Amy Chapman

I wish for every rare person to have a medication or a cure.

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Jenni Ward

I would like to see a change in how our Social Security Administration evaluates disability.

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Rebekah Palmer

A big difference to me would be more assistance in occupation as an adult living...

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Kim Madden

Rare disease research should be a relay race where we're all carrying the same baton.

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Megan Freeman

I would love to see if there could be another clinical trial done for my...

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Ruth Hochheiser

Education of the medical community about Orthostatic Tremor, including primary care physicians and neurologists.

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Christine McGarvey

Streamline the diagnostic odyssey by improving awareness of rare diseases among the medical community.

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Raymond A Huml

I want more robust incentives for companies developing rare disease drugs, especially for ultra-rare diseases.

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Alicia Dwyer

I have Susac Syndrome. We need research, as not one organization is doing it.

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Tytina Sanders-Bey

Access to care and treatment for specific health challenges. Easier access to prescription medication. Perhaps...

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Tammy Jones

My vision is to make connections with Rare Patients, one way I believe I can...

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Lesley Bennett

Expand Newborn Screening (including rare epilepsies) and establish Rare Disease Advisory Councils in every state.

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Sofia Horan

I hope to tell more patients that there is a treatment and there is hope.

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Jhoanny Cardenas

It would be wonderful if there was more focus on Rare Diseases for medical students.

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Cindy Choudhary

I would reimagine a healthcare system that could help treat my illnesses and actually listen.

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Jeneva Stone

I am hopeful that Maryland, my state, will establish a Rare Disease Advisory Council this...

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Janet Wilson

If there’s a way for more families with the same genetic results to submit to...

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Kerri Engebrecht

My passion and my plea is for mental health care to be looked at seriously...

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