Firefly Fund

03/02/2026

Last week, the Zevra team had the honor of attending the Firefly Dreams Gala, a powerful evening dedicated to supporting rare disease research and education, specifically Niemann-Pick disease type C (NPC).

This year’s cowboy chic theme brought energy and heart to an unforgettable night. Most importantly, it was a moving reminder of why raising awareness for rare diseases like NPC matters so deeply.

We’re proud to stand alongside this community and support the mission.

02/27/2026

In recognition of Rare Disease Awareness Month and Rare Disease Day (Feb 28th) Firefly Fund honors the more than 300 million people across the world living with a rare disease (National Institutes of Health).

We stand united with rare disease patients, families, clinicians, researchers and advocates supporting early diagnosis and those advancing research and life-changing, life-saving treatments.

Learn more about our core programs serving the community – patients, families and clinicians – with essential resources and support, advancing knowledge about the complexities of rare diseases: https://fireflyfund.org/our-programs/

02/22/2026

Firefly Dreams brought together an incredible community of supporters celebrating our 10 year anniversary. A Texas-sized shout-out with heartfelt gratitude to our guests including families, who traveled from across the country, our sponsors and team who brought this special evening to life.

New to Firefly Fund? Get involved! Connect with us here and online at fireflyfund.org



Emcee: Lauren Petrowski
Auctioneer: David Ackel
Music: Jonathan Terrell
Auction Platform: HelloFund
A/V Support: Robby Robinson

Event Committee
Stacy Turlington Dollar
Jennifer Johnson
Suzanna Rosenblatt
Kelly Gameson Breeden

Firefly Fund Co-Founders
Pam Crowley Andrews
Chris Andrews

Firefly Fund Staff
Valerie Beesley
Ryan Junell
Emma Andrus

Sponsors
Mandos by Beren Therapeutics
IntraBio Inc
Zevra Therapeutics
Lara Keel
Laura & Kyndel Bennett
Cyclo Therapeutics, Inc.
SEMbio
Stacy & Mike Toomey
Elizabeth Crook & Mark Lewis Foundation
John & Kelly Breeden
Westbank Flower Market
Mark Rowe Henna Chevrolet
The Garden Room
Randy & Stan Schleuter
Kori A. & Brian J. Phenegar
David & Sude Hart
Monamine Oxidase Deficiency Foundation
Texas Children's Hospital
Susie Dudley & Sal Conti
AlphaRose Therapeutics
Allen Boone Humphries Robinson LLP

02/15/2026

💛💛💛 Love to our amazing Firefly Dreams Platinum Sponsors — LLK LLC, Cyclo Therapeutics, and Laura & Kyndel Bennett — for helping this night shine brighter.

Cyclo Therapeutics, a Rafael company
cyclotherapeutics.com

LLK LLC
llkllc.com

02/14/2026

Our upcoming Firefly Dreams gala is only possible with the support of our generous sponsors. 💫 We’re grateful to presenting sponsor, Mandos® by Beren Therapeutics, a Public Benefit Corporation.

Mandos® is a biotechnology company committed to the development of adrabetadex for individuals living with Niemann-Pick Disease Type C (NPC), a condition characterized by defects in cholesterol trafficking.

Learn more
https://mandoshealth.com

02/14/2026

We’re so grateful to our presenting sponsor Zevra Therapeutics for helping bring our annual gala Firefly Dreams to life. ✨

Zevra is a rare disease therapeutics company with therapies approved in the U.S. for the treatment of Niemann-Pick disease type C (NPC).

Learn more
https://zevra.com

02/14/2026

We’re deeply thankful to the sponsors who make Firefly Dreams possible—especially our presenting sponsor IntraBio for helping to light the way. 💛

IntraBio is a biopharmaceutical company focused on discovering, developing, and commercializing therapies for neurodegenerative diseases with high unmet medical needs.

Learn more
https://intrabio.com

02/12/2026

We’re thrilled to count down to Firefly Dreams, our annual gala where we will celebrate and reflect on 10 years of our community coming together.

Learn more about our mission to accelerate cures for rare neurodegenerative genetic diseases that affect children at FireflyFund.org

12/19/2025

Our village has helped us accomplish everything we’ve achieved - this past year, and for almost 10 years since Belle and Abby were diagnosed.

What we’re continuously reminded of is that our village is here to save lives, and unlock the impossible.

What we’re accomplishing together is monumental, and we’re so grateful to you all.

12/17/2025

The Pediatric Priority Review Voucher (PPRV) program’s authorization lapsed a year ago, but advocates hope Congress will pass reauthorization before year’s end.

“What’s at stake is a bunch of kids’ lives, including the lives of my daughters Belle and Abby Andrews,” said Pam Crowley Andrews, Executive Director and Co-Founder of the Firefly Fund.

“Without the vouchers, it’s not realistic or viable for these drugs to make their way through the regulatory process. These vouchers provide real support that is critical for drug development for the ultra rare disease population.”

Read the full article

Patient advocates discussed one of the most important factors enabling breakthroughs against rare diseases, the PPRV, at a BIO coffee chat.

11/18/2025

🔬 Families experiencing pediatric neurodegenerative diseases like Niemann Pick Type C face a difficult race against time. This disease progressively steals children's abilities – their movement, speech, and ultimately their lives. The hashtag in H.R.1 protects critical incentives that accelerate the pace of research for these fatal diseases, providing hope needed to fuel families awaiting progress when every moment counts.

🏛️ This legislation dismantles a barrier that disincentivized drug development for ultra-rare pediatric conditions. By protecting crucial incentives for pharmaceutical companies to invest in rare disease research, Congress has empowered researchers to pursue treatments for conditions that affect smaller populations but devastate every family they touch. The hashtag fundamentally reshapes what's possible for the most vulnerable patients.

👇 Pam Crowley Andrews, Executive Director of Firefly Fund, knows the urgency of this fight firsthand. Her powerful message below captures why this law represents more than policy reform – it's a critical accelerator that could mean the difference between breakthrough and heartbreak for families racing against fatal diagnoses.

🔬 Families experiencing pediatric neurodegenerative diseases like Niemann Pick Type C face a difficult race against time. This disease progressively steals children's abilities – their movement, speech, and ultimately their lives. The in H.R.1 protects critical incentives that a...