Firefly Fund provides crucial support for research and education necessary to accelerate cures for rare genetic diseases that affect children and currently have no cure. The inspiration behind the Firefly Fund came after parents Pam and Chris Andrews discovered that both of their daughters, Belle age 6 and Abby age 2, had Niemann-Pick Type C1 (NPC1) in early 2016. NPC1 is a progressive neurodegenerative disease that attacks every cell of the human body and has a lifespan of 12-20 years.
Firefly Fund的外部链接
P.O. Box 92616
US，Texas，Austin，78709
Firefly Fund转发了
Zevra Therapeutics deeply appreciates the work of #NiemannPick advocacy groups such as the Firefly Fund for their dedication to improving the lives of those affected by #NPC. Thank you for your unwavering support of the NPC community. #NiemannPickAwareness
Firefly Fund转发了
"The FDA has to listen to the advocacy groups...because frankly, the advocacy groups have more experience than the FDA in the drug and in the disease." -Chris Andrews, founder of Firefly Fund, on this week's podcast
Thank you Jenna King and Fox 7 Austin for shining a light on NPC, the new FDA approved drugs, and on the Andrews family journey. #shiningalightonraredisease #fireflyfund #niemannpick
Pam and Chris Andrews joined Jason Scharf's podcast, Austin Next, to chat about Firefly Fund, our hopes for the #CenterforRareDisease at the Dell Medical School at the University of Texas at Austin and how #Austin is at the forefront of the intersection of #tech and #healthcare. Grab a cup of coffee and have a listen. Thank you, Jason, for believing in our mission and helping us #ShineALight on how the rare disease community can help light the way for the future of precision medicine in Austin, in #Texas, and in the world. #fireflyfund #ShiningALightOnRareDisease #JoinTheFight #NewbornSreeningSavesLives
Nothing motivates a parent to action like a sick child. Words like "no" & "impossible" are merely roadblocks & on today's Austin Next we learn what that means for Austin & the world. I'm joined by Pam Andrews & Chris Andrews, founders of the Firefly Fund, who turned their battle with their daughters' rare genetic disorder into a mission to revolutionize rare disease research & treatment. We discuss: -Firefly Fund's Origin -Establishing a Rare Disease Center at UT Dell Medical School? -Navigating the Diagnostic Odyssey -Interplay of Tech Innovation and New Business Models in Healthcare -Downstream Effects of Rare Disease Advancement The Andrews' vision? Austin at the forefront of tech & healthcare innovation. Soon, people will think of Austin for UT, BBQ, & saving lives! ?? Catch the full episode on all podcast platforms. Links in comments below!? What's Next?
Thank you, Nicole VILLALPANDO and Austin American-Statesman for covering the excitement in the NPC community around the FDA approvals of Miplyffa + Miglustat and Aqneursa. And an extra thank you for shining a light on Firefly Fund's dedication to advancing these drugs into clinical use and integrating NPC into the U.S. healthcare system over the years. It takes a village! #newbornscreeningsaveslives #NPCCommunity #shiningalightonraredisease #JoinTheFight #austin #atx
Today is the last day of Newborn Screening Awareness Month! Newborn screening (NBS) is a public health program of screening in infants shortly after birth for conditions that are treatable, but not clinically evident in the newborn period. The goal is to identify infants at risk for these conditions early enough to confirm the diagnosis and provide intervention that will alter the clinical course of the disease and prevent or ameliorate the clinical manifestations. Firefly Fund advances efforts to add rare diseases to newborn screening lists at state and federal levels. With your help, we can make a difference! #newbornscreening #pregnancy #raredisease #newbornscreeningawarenessmonth
Thank you Jason Scharf for the great conversation. We look forward to sharing the podcast episode soon.
Just wrapped recording an inspiring Austin Next episode with Pam Andrews & Chris Andrews of Firefly Fund. We discussed their mission to tackle rare disease, the need for both scientific invention & business innovation in health, and how rare disease research could unlock treatments for Alzheimer's and beyond. The story of their daughters odysseys reminds me why I personally drive for innovation in Bio & Health. There are lives on the line! Episode drops next week!
Thank you to the team at #IntraBio! What a week we have had. #keepdreaming #fireflyfund #jointheflight
Thank you to the small AND mighty team at IntraBio Inc, headquartered right here in ATX, for bringing #AQNEURSA to #NPC patients. What a remarkable testament this approval is to the power if #collaboration and #partnership. Over the past six years, Firefly Fund has had hundreds of bi-weekly calls with IntraBio Inc working side-by-side to ensure that US #NPC patients are able to access this therapeutic option, which has been indicated for the neurological manifestations of #NPC. @ChrisAndrews and I can NOT wait to add this therapy to our arsenal for Belle & Abby. Let's raise a glass to more tomorrows. Next up... Newborn Screening for NPC - were going straight for the #RUSP. #fireflyfund #shiningalightonraredisease #newbornscreeningsaveslives #jointheflight
Next week (9/26) we host Firefly Dreams – a benefit in support of rare disease research and education. RSVP to join the guest list https://lnkd.in/gwDHTpnY Enjoy food, drinks, and live music performances by Monte Warden and Eddie Ray Band! Monte Warden (w/ Kelly Willis on Austin City Limits) https://lnkd.in/gcNB9RGZ Eddie Ray Band (promo video) https://lnkd.in/gqA3TfWy