Heather Gatcombe, MD

YouTube video by All Access DNA Can patients with mitochondrial disease afford to wait for the FDA? Episode 33

You cannot diagnose mitochondrial disease or dysfunction by appearance

We discussed the importance of genetic testing, increasing awareness of mitochondrial diseases, potential loss of access to elamipretide, ❤️ transplant journey & resources for patients and caregivers

youtube.com/watch?v=V8RO...

Join the #ShotsForShotsChallenge—to fight for every patient’s shot at life.

💚 This is for every child fighting mitochondrial disease.

💚 For every baby with heart failure whose life depends on elamipretide.

Patients may lose access to this medication as soon as September
#APPROVEELAMIPRETIDE

@ossoff.senate.gov @ossoff.bsky.social @warnock.senate.gov @washingtonpost.com @wsj.com @nytopinion.nytimes.com @nytimes.com @cnn.com @npr.org @usatoday.com @todayshow.com @fda.gov @ajc.com @theatlantic.com

@nytimes.com @ossoff.senate.gov @ossoff.bsky.social @warnock.senate.gov @fda.gov @axios.com @postopinions.bsky.social @usatoday.com @wsj.com

@umdfg.bsky.social

My son’s mitochondrial disease led to heart failure and a transplant.

No family should have to experience that journey.

Join the #ShotsForShotsChallenge to fight for every patient’s shot at life

💚 For every baby with heart failure whose life depends on elamipretide

Wall Street Journal reported this week on how the FDA is failing patients with rare diseases.

Babies with heart failure may lose the life saving drug Elamipteride as soon as next month.

How the race to invent a drug for one sick baby made medical history A rare disorder threatened baby KJ. Could a sprint to edit his genes using CRISPR save him?

wapo.st/3Hd4b9S. This story offers so much hope to families of children with rare and metabolic diseases. ❤️💕🥰

Such a heartening story