Time is running out for kids with a rare disease, parents say, unless the FDA acts After a more than decadelong process to bring the experimental drug elamipretide to market, the treatment’s approval has repeatedly hit roadblocks.

“Our kids are dying. We have seen that this drug works,” said Kate McCurdy, a co-founder of the Barth Syndrome Foundation. “This drug totally saves the lives of babies.”

#elamipretide #ApproveElamipretide #FDAActNow #BarthProgress #BarthSyndrome #RareDisease #MitochondrialDisease

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

#ApproveElamipretide
#FDAActNow #BarthProgress #Barthsyndrome #RareDisease #MitochondrialDisease #Mito #shotforshots @fda.gov

#BarthProgress
#ApproveElamipretide
#BarthSyndrome
#RareDisease
#FDAActNow
#HopeForBarth

#isolation #cancersurvivormd #primarymitochondrialdisease #raredisease #approveelamipretide #fdaactnow #hopeforbarth #barthprogress #shame #blame @susancain.bsky.social @stonybrooku.bsky.social

#BarthProgress, #ApproveElamipretide, #BarthSyndrome, #RareDisease, #FDAActNow, #HopeForBarth

#BarthProgress
#ApproveElamipretide
#BarthSyndrome
#RareDisease
#FDAActNow
#HopeForBarth
@annabower.bsky.social @muellershewrote.com @fda.gov

#ApproveElamipretide #HopeForBarth #RareDisease #FDAActNow #Barthsyndrome