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

We’re taking part in productive and impactful #HillMeetings today. On behalf of our #BarthSyndrome community, we’re telling our stories and sharing our desperate need for a treatment. #NotTooRareToCare #RareDC2024 #RareDiseaseDay #RareDiseaseWeek #RareDisease

Thank you, Senator Mike Braun, for listening to our perspectives as #BarthSyndrome patients, caregivers & advocates. Please hold U.S. FDA accountable to review the data for the only potential medicine for our life-threatening #RareDisease. #RareDiseaseDay #NotTooRareToCare #RareDC2024

We’re at The White House! Randi Clites #DrJanetWoodcock #RareDC2024 #RareDiseaseDay2024

“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 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

#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

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 #RareDiseaseWeek. Hold U.S. FDA accountable for fair reviews of #RareDisease drugs. #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, Senator Mike Braun, for your support of #RareDisease patients! We appreciate the chance for #BarthSyndrome advocates to meet you during #RareDiseaseWeek and share more about our desperate need for a treatment. #NotTooRareToCare #RareDC2024

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

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…

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

Excellent reporting of the current situation by ed silverman Barth Syndrome

We are at a decisive moment where #Duchenne is just beginning to evolve from a death sentence to a manageable chronic condition. The U.S. FDA #acceleratedapproval pathway is key to seizing this moment and saving lives. More in STAT today.

Exciting update! U.S. FDA has now determined that the New Drug Application for elamipretide to treat #BarthSyndrome will receive a Priority Review designation. Thank you FDA for recognizing the serious and unmet medical needs of our #BarthSyndrome community. Read more:

Join us for Barth Syndrome Foundation’s virtual townhall on May 23rd at 12pm ET to learn more about the valuable role that the worldwide Barth syndrome community will play in the FDA’s review of Stealth’s new drug application (NDA) for elamipretide. Register here:

.Paul Melmeyer: "There are a number of flexibilities —to give U.S. FDA a shout-out — the use of accelerated approval, especially within the center for biologics, what we're seeing there, the direction of using accelerated approval for gene therapy as we saw this with Duchenne last

IGT urges #Congress & other stakeholders to improve access to #newbornscreening to help patients receive the gene therapies they need. Thank you Lesa Brackbill Gaucher Community Alliance Jennifer Handt Erik Paulsen for raising your voices! BLOG: bit.ly/IGT-NBSAwarene… #NBSAwarenessMonth