“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
#elamipretide #ApproveElamipretide #FDAActNow #BarthProgress #BarthSyndrome #RareDisease #MitochondrialDisease
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.
bit.ly
August 19, 2025 at 3:29 PM
“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
#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
💚 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
August 17, 2025 at 1:55 PM
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
💚 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
#FDAActNow #BarthProgress #Barthsyndrome #RareDisease #MitochondrialDisease #Mito #shotforshots @fda.gov
August 14, 2025 at 10:24 PM
