Firefly Fund

10/13/2026

NPC is a rare genetic neurodegenerative disease that is estimated to affect only 1 in 150,000 people. NPC belongs to a larger group of more than 100 disorders known as lysosomal storage disorders. Patients with NPC disease are unable to make functional NPC protein, which is used by cells in the body to recycle cholesterol and lipids through the lysosome to other parts of the cell. The absence of functional NPC protein causes a toxic accumulation of cholesterol and other lipids in the lysosomes, while other parts of the cell are starved. This results in cell death and leads to irreversible organ damage.

Learn more about NPC: https://fireflyfund.org/about-npc1/

10/10/2026

The Firefly Fund was inspired by the NPC diagnosis of Pam and Chris Andrews’ daughters. The organization was founded with a vision and mission of ending the long diagnostic odyssey experienced by rare disease families and to change what is possible for NPC patients and caregivers by accelerating cures for children facing a rare disease diagnosis.
Learn more about our founders’ story: https://fireflyfund.org/about-us/our-story/

10/09/2026

October is NPC Awareness Month, a global initiative that aims to bring more awareness to Niemann-Pick diseases including Niemann Pick Type C (NPC), an ultra-rare, progressive, and neurodegenerative genetic disorder.

We’re honored to stand with the international Niemann Pick Disease community of clinicians, researchers, patient advocates and pharmaceutical leaders working to advance promising treatments for NPC.

Learn more about NPC: https://fireflyfund.org/about-npc1/

09/28/2026

Why is newborn screening so important?

There are more than 7,000 different known rare diseases. Roughly one in 10 or 30 million Americans live with a rare condition.

As part of our mission Firefly Fund works to drastically shorten and help end the long diagnostic odyssey typically experienced by families with a rare disease. and are both crucial diagnostic screening tools used to identify conditions early, before visible signs or symptoms, so patients can intervene early. Catching these neurodegenerative diseases early drastically improves health outcomes and can change the trajectory of these patients’ lives.

Learn more from NPC researcher Dr. Dan Ory on the vital importance of early detection and treatment of NPC:

https://www.youtube.com/watch?v=iayvxnDxec0

Dr. Daniel Ory explains how newborn screening can save years of hea...

09/27/2026

Firefly Fund is steadfast in our efforts advocating to ensure NPC is added to the federal Recommended Uniform Screening Panel (RUSP) that guides state newborn testing.
Help us get NPC added: https://fireflyfund.org/contact/

09/26/2026

Sharing again with the link in case you missed it: Throwback Thursday: Throughout 2026, we’re celebrating Firefly Fund’s 10 year anniversary. Throwback to our truly memorable Firefly Dreams gala evening and the impactful voices from our program. Thank you for joining us and sharing your lived experiences.

We are so grateful for the energy, enthusiasm and heartfelt contributions from our community! A Texas-sized shout-out again for these memories - to guests including families, who traveled from across the country, our sponsors and team who brought this special evening to life to with Firefly Fund. Cheers to all of you!

Watch our highlights video here:

Relive the highlights from Firefly Dreams Gala 2026, Firefly Fund’s...

09/26/2026

An incredible strength of our patient community is our support for one another across geographies. Our community is our lifeline in so many ways.

Do you have an NPC journey to share? Reach out with your story here: https://fireflyfund.org/share-your-story/

Firefly Fund invites you to share your own rare disease journey to help us shine light on rare genetic diseases that affect children and currently have no cure. To submit your story, please use the form at the bottom of this webpage. For questions or more information, contact [email protected]

09/25/2026

This month, and every day of the year, we are committed to advancing newborn screening. Early intervention matters. Knowing sooner is better. Learn more at fireflyfund.org

09/25/2026

saves lives. Join us in recognizing advocates working tirelessly to ensure devastating diseases like NPC are added to newborn screening lists in every state. Learn more at fireflyfund.org

09/24/2026

Throwback Thursday: Throughout 2026, we’re celebrating Firefly Fund’s 10 year anniversary. Throwback to our truly memorable Firefly Dreams gala evening and the impactful voices from our program. Thank you for joining us and sharing your lived experiences.

We are so grateful for the energy, enthusiasm and heartfelt contributions from our community! A Texas-sized shout-out again for these memories - to guests including families, who traveled from across the country, our sponsors and team who brought this special evening to life to with Firefly Fund. Cheers to all of you!

Watch our highlights video here: