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R McCarthy

@reeniemccarthy
 NE
Joined November 2014
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Photo by R McCarthy (@reeniemccarthy)Photo by R McCarthy (@reeniemccarthy)Photo by R McCarthy (@reeniemccarthy)Photo by R McCarthy (@reeniemccarthy)Photo by R McCarthy (@reeniemccarthy)Photo by R McCarthy (@reeniemccarthy)
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
Image from Barth Syndrome (@BarthSyndrome)
Image from Barth Syndrome (@BarthSyndrome)
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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
Image from Barth Syndrome (@BarthSyndrome)
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“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
Image from Barth Syndrome (@BarthSyndrome)
Image from Barth Syndrome (@BarthSyndrome)
Image from Barth Syndrome (@BarthSyndrome)
Image from Barth Syndrome (@BarthSyndrome)
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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
Image from Barth Syndrome (@BarthSyndrome)
Image from Barth Syndrome (@BarthSyndrome)
Image from Barth Syndrome (@BarthSyndrome)
Image from Barth Syndrome (@BarthSyndrome)
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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 energycommerce.house.gov/pos…
Chairs Rodgers and Guthrie Announce Health Subcommittee Legislative Hearing on Rare Disease Bills

Chairs Rodgers and Guthrie Announce Health Subcommittee Legislative Hearing on Rare Disease Bills

The Committee on Energy and Commerce is the oldest standing legislative committee in the U.S. House of Representatives and is vested with the broadest jurisdiction of any congressional authorizing...

energycommerce.house.gov
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“#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
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"At the current pace, it will take thousands of years for all #RareDiseases to have treatments." @KhrystalKDavis @EnergyCommerce #RareDiseaseDay #NotTooRareToCare #RareDC2024
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"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
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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 nottooraretocare.org/
Image from Barth Syndrome (@BarthSyndrome)
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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
Image from Barth Syndrome (@BarthSyndrome)
Image from Barth Syndrome (@BarthSyndrome)
Image from Barth Syndrome (@BarthSyndrome)
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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
Image from Barth Syndrome (@BarthSyndrome)
Image from Barth Syndrome (@BarthSyndrome)
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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
Image from Steve Graessle (@McLovin9886)
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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
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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
Image from RARE Foundation (@RareAdvocates)
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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
Image from Barth Syndrome (@BarthSyndrome)
Image from Barth Syndrome (@BarthSyndrome)
Image from Barth Syndrome (@BarthSyndrome)
Image from Barth Syndrome (@BarthSyndrome)
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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.
Barth Syndrome (@BarthSyndrome) profile pictureBarth Syndrome@BarthSyndrome21 Dec 2023
Today we delivered a petition to the FDA that garnered nearly 20,000 signatures FDA, we expect a response and definitive action for Barth syndrome patients and families! prnewswire.com/news-releases…
Kate McCurdy, board chair of Barth Syndrome Foundation, poses for a portrait, while holding a petition with nearly 20,000 signatures requesting the U.S. Food and Drug Administration (FDA) to review the New Drug Application (NDA) for elamipretide, the only potential treatment for Barth syndrome, at the FDA White Oak Campus in Silver Spring, Maryland, USA, on Thursday, December 21, 2023. (Barth Syndrome Foundation/Eric Lee)

ALT Kate McCurdy, board chair of Barth Syndrome Foundation, poses for a portrait, while holding a petition with nearly 20,000 signatures requesting the U.S. Food and Drug Administration (FDA) to review the New Drug Application (NDA) for elamipretide, the only potential treatment for Barth syndrome, at the FDA White Oak Campus in Silver Spring, Maryland, USA, on Thursday, December 21, 2023. (Barth Syndrome Foundation/Eric Lee)

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Dive into the fascinating world of cellular dynamics and discover the pivotal role of #mitochondria in #AMD with this informative article: bit.ly/3EvKgym 👀🤓 #Sponsored
Image from Retinal Physician (@RetinalPhys)
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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: ow.ly/Sk7A50O0YGh
Image from Barth Syndrome (@BarthSyndrome)
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Thank you to Congressmen @RepPaulTonko (D-NY), @RepGusBilirakis (R-FL) and @RepRalphNorman (R-SC) for your leadership in introducing this bipartisan legislation to establish April 5th as Barth Syndrome Awareness Day.
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We're joining the @BarthSyndrome Foundation to recognize & celebrate the first Barth Syndrome Awareness Day! We celebrate this day as a call-to-action to make therapies possible & accessible for patients with Barth syndrome. Learn how you can support here: bit.ly/3zzTjfm
Image from Mighty Therapeutics (@Mighty_Tx)
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