Abe's Story

Abe is one of just 130 children and young adults in the U.S. (and less than 250 people worldwide) currently living with Barth syndrome, an ultra-rare, life-threatening, genetic disease. We are honored to share this family’s video highlighting their personal experience and the hope they hold for Abe’s future.
#BarthSyndrome, #raredisease, #clinicaltrials, #FDA, #BarthStrong, #GeneticDisease, #ProfilesInCourage, #AbesStory, #PatientVoices, #ultrararedisease, #advocacy, #TeamBarth, #TreatmentChoices, #TreatmentOptions, #elamipretide, @USFoodandDrugAdmin

[@cmjohnsson]

Abe is a good friend to my son. Kind hearts like his make the world a better place. 🥰

[@mrmobileroblox5115]

Very touching

[@alannaboozer5901]

This was so well done; thank you so much for sharing your story! #BarthStrong #FDA #ClinicalTrials #raredisease

[@helencosta4234]

What a sweet articulate brave little boy. Thanks for sharing your story, Abe.

[@stefangirgenrath5360]

That's a great story. Abe and his family, don't get bogged down by the disease. You all are reaching for the stars and I see some shimmer of hope that there is a treatment option coming soon if it is getting approved by the FDA.

[@anitamyers856]

Thank you Abe and your parents for this great video that advocates for all Barth boys and their families. You did a great job! Pray for positive decision from FDA

[@heimendonker7732]

Essential for many. A 'must have' therapy!

[@moshiesty9882]

Hopefully fda can accept NDA submission , so little sweet souls like Abe can have a fighting chance at life

[@chambrick3043]

Thanks for sharing your story with us ❤ praying for advancements in science 🙏

[@fullback118]

Thank you so much Abe and family for sharing your story. Here's to hoping for a favorable decision by the FDA regarding the use of elamipretide to treat Barth syndrome.

[@TheKatarinaGiselle]

Can we have an Abe update? I know they are doing SO much in stem cell therapy, especially with Barth syndrome. Have they looked into that?

[@oregondeanRARE]

Abe's story of the story of hundred with Barth Syndrome and 30 million with one of the other 7,000 Rare Diseases. Yea - what about the "risk " of a great outcome - let's seize it! #FDA #RareDisease #MLD #FMLD

[@mrmobileroblox5115]

😊

[@Clemburke1111]

My nephew has this