I am writing with profound urgency regarding the approval of Elamipretide, a life-changing medication for individuals diagnosed with Barth syndrome. This includes my three-month-old grandson, Giancarlo Natale.  

  When G was born, his parents were told he was healthy. However, within weeks, they noticed alarming changes — his breathing patterns, color, and an overall decline. At just a few weeks old, he suffered acute heart failure, his tiny heart losing nearly all function. 

  Further testing revealed that he was one of only 300 individuals worldwide diagnosed with Barth syndrome, an ultra-rare mitochondrial disease that prevents his body from producing the energy necessary for his muscles, including his heart, to function properly. 

  Their greatest hope arrived in the form of Elamipretide, a study drug that was granted to G through emergency access. This medication tightens his mitochondria, providing his body with the energy needed for survival and growth. Thanks to Elamipretide, G has made extraordinary progress. However, its continued availability is now in jeopardy. 

  Stealth BioTherapeutics, the pharmaceutical company behind Elamipretide, has fought relentlessly to keep this medication accessible despite financial strain. They have put patients above profit, but without FDA approval, they may not be able to continue providing this life-saving medication. 

  Recently, the FDA denied their application, forcing them to resubmit, but this resubmission could exclude the very infants whose lives depend on Elamipretide. G is one of those babies. 

We urge you to recognize the critical need for Elamipretide’s immediate approval for all Barth syndrome patients. Delaying its approval endangers lives and robs individuals like G of their best chance at a future. 

  I implore you to reach out to the FDA to insist on the approval of Elamipretide so that every person battling Barth syndrome can continue receiving the treatment they desperately need. Their survival depends on it. 

  Peggy Bradley,  Chestnut Hill