Next week I am going to Washington D.C. to advocate for a fair and equitable U.S. FDA review of the only investigational medicine for Barth syndrome, the life-threatening #RareDisease that claimed the lives of my sons. #RareDisease Day #NotTooRareToCare nottooraretocare.org

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

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

“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

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

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

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

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

Thank you, Paul Tonko, for advocating on behalf of #RareDisease communities for consistency in regulatory reviews of much-needed investigational treatments & for highlighting the unmet needs in #PraderWilliSyndrome . We appreciate your letter to U.S. FDA: nottooraretocare.org/wp-content/upl…

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

It was an honor to join #BarthSyndrome advocates for #RareDisease week

#RareDC2024 #NotTooRareToCare
#RareDisease Day

Visit nottooraretocare.org to learn more and join us in showing our stripes, letting this community know they are not alone in their journey.

#RareDiseaseDay #ShowYourStripes

#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

Research article from Portugal #smas #ncs #raredisease #medtwitter #nottooraretocare #zebra #vascularcompressiosyndromes scielo.br/j/jvb/a/HVDbgC…

New Zealander needs to travel to Germany for surgery. ⁦Medical Council of New Zealand⁩ why must your people fundraise for these #lifesaving surgeries #raredisease #help #nottooraretocare givealittle.co.nz/cause/health-s…

Terminal Tower CLE Thank you! #smas2024 #nottooraretocare

Today as I was following a twitter thread I saw this 😢Elliot K Fishman / CTisus.com National MALS Foundation NORD Easton Allred #nottooraretocare #smas #raredisease #zebra

Interesting use of stent to solve complex #SMAS patient needs UNC-Chapel Hill Always happy to see #surgery thinking outside the box for patients! #NotTooRareToCare #RareDisease #zebra #smasyndrone amjcaserep.com/abstract/full/…

Another child diagnosed with #SMAS We continue to add patients to our online communities while doctors continue to dismiss this syndrome as too rare. #MedTwitter #GITwitter #surgery #raredisease #NotTooRareToCare Kaehlers call on community wahpetondailynews.com/news/kaehlers-… via Wahpeton Daily News Media