For the 95% of rare disease patients still without an approved treatment, FDA is working for you. Acting Commissioner Kyle Diamantas shares how FDA is transforming rare disease drug development through the new Rare Disease Framework Initiative. Because every family deserves answers, action, and hope. fda.gov/news-events/fda-voic…
Delivering Answers and Hope for Rare Disease Families

Delivering Answers and Hope for Rare Disease Families

Rare diseases present our most formidable challenge and our greatest opportunity to speed life-saving treatments and cures to the patients who need them most.

fda.gov

Nov 5, 2026 · 5:08 PM UTC

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Baby KJ’s case is really inspiring. Base editing and prime editing will play a key role in helping many patients with rare diseases.
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Thank you, @FDA_KyleD and @FDA, for recognizing the urgency facing rare disease families and advancing a framework that reflects the complexities of diseases like Huntington’s disease. The HD community has contributed decades of natural history data, clinical trial participation, and documented risk tolerance. We welcome Priority Review of AMT-130’s BLA, but continue to advocate for CNPV consideration. If a promising gene therapy for a fatal, relentlessly progressive disease does not warrant consideration for every appropriate expedited pathway, what does? The success of regulatory reform will be measured not by the hope it promises, but by the patients it reaches while there is still time. Our community has done its part. Now we look to the FDA’s leadership to ensure that urgency is translated into meaningful action. 💙💜 #HuntingtonsDisease #RareDisease
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Non-ambulent Duchenne Muscular Dystrophy boys have NOTHING to help them preserve upper body function. Until now. DERAMIOCEL. The FDA has the data - and it WORKS, obviously - 76% reduction in loss of upper limb function. That's MEANINGFUL - please approve it - STAT - every day they lose more muscle!
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No more lip service, please. Deramiocel from @Capricor is on your desk. Time to walk the walk or stfu about any new frameworks.
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"95% still without an approved treatment" is a wild stat to post when your one job is approving treatments.
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Would be nice if you actually created cures instead of stealing from the Medical sector as monopoly. 👽
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Approve #DCVAX for #GBM patients so they don’t have to travel overseas for treatment invented in the US! braintumourresearch.org/blog…
“Astonishing” results for brain tumour vaccine trial

“Astonishing” results for brain tumour vaccine trial

Read about the world’s first glioblastoma vaccine, the first major breakthrough in 17 years, and a new hope for patients with glioblastoma.

braintumourresearch.org
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CNPV for AMT-130. Every single day untreated for Huntingtons patients brings harm they can never reverse. The 4 month difference between AA and CNPV evaluation could be the difference for hundreds between being able to recieve lifesaving treatment or being too far gone to help.
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Deramiocel is waiting for a signoff at your desk for a while now. Don‘t delay. Time is precious.
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CAPR and the boys with DMD are waiting. Are you all talk or action?
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Wild stat when you have a safe and VERY effective therapy in Deramiocel. Approve it already! Sheesh!!
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Find cures for SCN2A gene mutations. We are not living in USA but we are waiting cure from you.
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NurOwn for ALS patients is still waiting for your approval. Somehow, it's taking you forever with no definite date.
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Hopefully you approve real drugs and not pbo drugs just for show and dance. FDA can’t get in a loop of approving drugs that don’t work and making incentives for companies to pursue that behavior.
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Deramiocel $CAPR
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Walk the talk and approve @Capricor deramociel product for DMD - it’s been nothing but lip service so far
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#dcvax #gbm #allhardtumors #jamaoncology #naturemay2024 #atldc #exvivo #flaskworks $NWBO #dcvaxforbraelyn #ukparliament #droz
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Deramiocel 🙏
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Meanwhile dmd patients continue waiting
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Why can't you guys keep all the crap that can harm us and one day kill us out of our food!!! Why do you allow any small amount of arsenic in our food? Why do you allow aluminum and other carcinogens in our vaccines? What is your guys's job again?
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Hurry up!
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#LongCovid #MECFS
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