News and Press Releases : News & Events : Barth Syndrome Foundation

Historic. I can’t help but think of my brothers and wish they were here to benefit from this. Mostly, I’m just so proud of my mom and everyone who worked so hard to make this happen. www.barthsyndrome.org/newsevents/n...

#BarthProgress #elamipretide #mitochondrialdisease #raredisease #barthsyndrome

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

#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
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