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 ultrarare, lifethreatening, 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