HAPPENING TOMORROW 4/26 at 12pm ET: How Loss of TAFAZZIN Function Ignites Mitochondrial Dysfunction

With special guests Miriam Greenberg, PhD; Valerian Kagan, PhD; Hülya Bayir, MD

Please contact Shelley Bowen for Zoom link and details: [email protected]

This year, I proudly reintroduced my resolution to recognize today as Barth Syndrome Awareness Day. Today and every day, I’m committed to raising awareness and bringing hope to the brave individuals and families impacted by this condition. congress.gov/bill/118th-con…

We have learned from Stealth BioTherapeutics that the U.S. Food and Drug Administration (FDA) has agreed to file and review the new drug application (NDA) for elamipretide for the treatment of Barth syndrome. The NDA, submitted by the drug’s sponsor Stealth BioTherapeutics, is a…

Joining us for today’s ceremonial puck drop is 8 year old Deacon and his family!

Deacon is a passionate, outgoing boy who was diagnosed with Barth Syndrome at 4 months old. He has been courageously fighting and continues to maintain a positive, engaged, and determined attitude.

Applications for On-site Research, Poster Submissions, and Travel Stipends are being accepted until March 29, 2024!

The Barth Syndrome Foundation welcomes applicants to submit proposals for on-site research, poster presentations, and accompanying travel stipends for the 2024…

Thank you Kristin Thorne for putting a spotlight on the urgency of a fair and equitable review by U.S. FDA of a potential treatment for #barthsyndrome , and to our families that continue to advocate for our community. abc7ny.com/barth-syndrome…

Myself and another NKY family affected by Barth Syndrome went to Washington for this fight. WCPO 9 Local 12/WKRC-TV FOX19 NOW WLWT Families of children with rare, life-threatening disease push FDA to review drug application abc7ny.com/barth-syndrome…

Families of children with rare, life-threatening disease push FDA to review drug application abc7ny.com/barth-syndrome…

Thank you, Senator Mike Braun, for your support of #RareDisease patients! We appreciate the chance for #BarthSyndrome advocates to meet you during #RareDisease Week and share more about our desperate need for a treatment. #NotTooRareToCare #RareDC2024

Thank you to the office of Leader McConnell for the chance to share that we are #NotTooRareToCare about. Hold U.S. FDA accountable for a fair review of the only investigational treatment for #BarthSyndrome . #RareDiseaseWeek #RareDC2024

We deeply appreciate the productive conversations with the offices of California representatives Mark DeSaulnier Nanette D. Barragán Judy Chu Rep. Juan Vargas Rep. Lou Correa during #RareDisease Week . Hold U.S. FDA accountable for fair reviews of #RareDisease drugs. #NotTooRareToCare

#BarthSyndrome advocates had the opportunity to advocate for a fair U.S. FDA review of the only investigational drug for our #RareDisease at the offices of Georgia Congressional leaders @Ossoff Senator Reverend Raphael Warnock Rep. Barry Loudermilk. Thank you for listening! #NotTooRareToCare #RareDC2024

Thank you to the offices of Iowa Congressional leaders, Chuck Grassley and Rep. Mariannette Miller-Meeks, M.D., for taking the time during #RareDiseaseWeek to learn more about #BarthSyndrome and our desperate need for a treatment. U.S. FDA #RareDiseaseDay #NotTooRareToCare #RareDC2024

Thank you, U.S. Senator Cindy Hyde-Smith, for meeting with #BarthSyndrome advocate Jacob during #RareDiseaseWeek and learning more about our community’s desperate need for a treatment. #NotTooRareToCare #RareDC2024

Last week, 20+ #BarthSyndrome advocates from 16 states participated in ~60 meetings with Congressional leaders in Washington DC. We advocated for U.S. FDA to listen to patients/caregivers & exercise fair, appropriate reviews of promising #RareDisease therapies. #NotTooRareToCare

Good night, DC! This was an empowering week. Pictured here is a young advocate who alongside his dad raised awareness for Barth syndrome
– a cause close to his heart, as both he and his brother live with it. #BarthSyndrome #rarediseaseweek #raredc2024 #nottooraretocare

The Barth Syndrome Foundation would like to thank Trevor from Rep. Morgan McGarvey office for meeting with us during Rare Disease Week in Washington D.C. on a fair and equitable FDA review process for rare drugs
#RareDiseaseDay2024 #NotTooRareToCare

“Wear comfortable shoes,” we were told. #BarthSyndrome is a disease characterized by fatigue & muscle weakness among other complications. Nevertheless we’re proud we walked miles in DC this week to advocate for much-needed treatment! #NotTooRareToCare #RareDC2024 #RareDiseaseDay

“ #RareDisease patients experience inequities in diagnosis and care. The majority of #RareDisease s do not have a U.S. FDA approved treatment.” Nanette D. Barragán Energy & Commerce Democrats #HealthEquity #RareDisease Day #RareDC2024 #NotTooRareToCare

Today is Rare Disease Day! A day when the rare disease community comes together to spread global awareness, raise our voices, and show our support to one another. Tag us in your social media posts today and tell us how you are celebrating Rare Disease Day 2024💚💗💙