R McCarthy

[Barth Syndrome @BarthSyndrome]

Thank you to the offices of Iowa Congressional leaders, @ChuckGrassley and @RepMMM, for taking the time during #RareDiseaseWeek to learn more about #BarthSyndrome and our desperate need for a treatment. @US_FDA #RareDiseaseDay #NotTooRareToCare #RareDC2024

[Barth Syndrome @BarthSyndrome]

Thank you, @SenHydeSmith, for meeting with #BarthSyndrome advocate Jacob during #RareDiseaseWeek and learning more about our community’s desperate need for a treatment. #NotTooRareToCare #RareDC2024

[Barth Syndrome @BarthSyndrome]

“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

[Barth Syndrome @BarthSyndrome]

We were honored to be at the @RareAdvocates @EverylifeOrg #RareDisease Congressional briefing. Thank you for all your efforts to amplify the voices of patient communities, including #BarthSyndrome, and show that we are #NotTooRareToCare about! #RareDiseaseDay #RDW2024 #RareDC2024

[Barth Syndrome @BarthSyndrome]

Thanks, @cathymcmorris and @RepGuthrie, for holding a hearing on legislation to support #RareDisease communities today at 10am ET. We’re looking forward to a productive discussion! #RareDiseaseDay #RareDC2024 #NotTooRareToCare https://t.co/dBzzZsW9A5

[Barth Syndrome @BarthSyndrome]

“#RareDisease patients experience inequities in diagnosis and care. The majority of #RareDiseases do not have a @US_FDA approved treatment.” @RepBarragan @EnergyCommerce #HealthEquity #RareDiseaseDay #RareDC2024 #NotTooRareToCare

[Barth Syndrome @BarthSyndrome]

"At the current pace, it will take thousands of years for all #RareDiseases to have treatments." @KhrystalKDavis @EnergyCommerce #RareDiseaseDay #NotTooRareToCare #RareDC2024

[Barth Syndrome @BarthSyndrome]

"For progressive #RareDiseases, any delay in establishing a treatment regimen means that there will be ground lost that can never be reclaimed." @michaelcburgess @EnergyCommerce #NotTooRareToCare #RareDiseaseDay #RareDC2024

[Barth Syndrome @BarthSyndrome]

Today is #RareDiseaseDay. More than 20 #BarthSyndrome advocates are in DC because there are no approved treatments for this life-threatening #RareDisease, & @US_FDA has refused to review the only investigational treatment. #NotTooRareToCare #RareDC2024 https://t.co/1cGEKfXU2m

[Barth Syndrome @BarthSyndrome]

20+ #BarthSyndrome advocates are sharing their stories on Capitol Hill & asking Congress to hold @US_FDA accountable to review the only potential medicine for this life-threatening #RareDisease. #NotTooRareToCare #RareDC2024 #RareDiseaseDay @RepRussellFry @RepEliCrane @RepDavid

[Barth Syndrome @BarthSyndrome]

Thank you, @SenatorBraun, for listening to our perspectives as #BarthSyndrome patients, caregivers & advocates. Please hold @US_FDA accountable to review the data for the only potential medicine for our life-threatening #RareDisease. #RareDiseaseDay #NotTooRareToCare #RareDC2024

[Steve Graessle @McLovin9886]

Next week I am going to Washington D.C. #RareDisease Week to advocate for a fair and equitable @US_FDA review of the only investigational medicine for Barth Syndrome, a life threatening condition I have.
@RepThomasMassie @RandPaul

[Barth Syndrome @BarthSyndrome]

In just 2 months, we garnered nearly 20,000 signatures on a petition advocating for a fair @US_FDA review of the only investigational medicine for #BarthSyndrome. Now we are in DC asking Congress to hold FDA accountable! #RareDiseaseDay #NotTooRareToCare #RareDC2024

[RDLA @RareAdvocates]

Capitol Hill, here we come!

These rare disease advocates are coming your way to make their voices heard.

Shout out to our virtual advocates and everyone who couldn’t make it in person. 🦓🙌

#RareDC2024 #legislativeconference #washingtondc #capitolhill

[Barth Syndrome @BarthSyndrome]

We’re so proud of our small but mighty #BarthSyndrome community, advocating in DC for a fair, appropriate @US_FDA review of the only investigational medicine for this #RareDisease. #RareDiseaseDay #NotTooRareToCare #RareDC2024

[Barth Syndrome @BarthSyndrome]

On behalf of the entire Barth syndrome community, we implore the FDA to review – in a fair, equitable, and appropriate manner – the only potential treatment for this life-threatening rare disease.

[Retinal Physician @RetinalPhys]

Dive into the fascinating world of cellular dynamics and discover the pivotal role of #mitochondria in #AMD with this informative article: https://t.co/x9Th5az9J2 👀🤓
#Sponsored

[R McCarthy @reeniemccarthy]

@DeannaPortero You should connect with Dr Hilary Vernon who runs one of only 2 Barth syndrome interdisciplinary clinics in the world at Hopkins. She is amazing, and Barth syndrome certainly fits your "too small to develop" criteria. Good luck - worthy cause

[Barth Syndrome @BarthSyndrome]

Recently, Lynda Sedefian, parent of two sons with Barth syndrome, Eric (who is deceased) and Derek, met with @RepPaulTonko [D-NY-20] to discuss his support of the Barth Syndrome Awareness Day Resolution in the US House of Representatives. Read more: https://t.co/pEa8q3WAWG

[R McCarthy @reeniemccarthy]

@KardinalLaw So incredibly sorry