@tadalper@1776AmericaNews@DC_Draino@instapundit@Capricor It is simple Math. The Deramiocel Phase 2 and Phase 3 results are stellar. Check the p values. The patients and their families are rightfully asking for this DMD treatment.
@DC_Draino@DrMakaryFDA owes it to the American people to correct the unforgivable mistakes Vinay Prasad has made! The patients and their families waiting for the approval of drugs for rare diseases have suffered needlessly for too long!
@DrMakaryFDA@Capricor has stellar Phase 3 results. Dr. Makary, time to walk to talk and approve Deramiocel. The patients and their families have been patiently waiting for this treatment. @RobertKennedyJr@Elijahjstacy
The Han Phan letter in Anthony's thread was neither related to @Capricor nor its Hope trials. I hope the company's legal team looks into these claims. $CAPR
The Han Phan letter in Anthony's thread was neither related to @Capricor nor its Hope trials. I hope the company's legal team looks into these claims. $CAPR
๐งต$CAPR longer thread, full report available now on substack, link in bio.
in this thread I'll get into more of the details of why CAP-1002 is about to fail its ph3 topline
@elonmusk@LauraLoomer Prasad blocked the DMD Cure Drug whose study had a p value of 0.0066.
Please read Elijah's Mom's heartfelt open letter to @realDonaldTrump
President Trump โ my momโs greatest desire has always been to see her kids get better. Even while battling Stage IV pancreatic cancer, she is still fighting for that day.
Please read her letter to you. I ask you to intervene, as you have before, to cut through the red tape and help save my life โ and the lives of thousands living with Duchenne Muscular Dystrophy.
Please share this. We need every voice.
@realDonaldTrump@WhiteHouse@JDVance
https://t.co/noniyn8bOh
@Elijahjstacy@realDonaldTrump@VP@SecKennedy
Stacy's letter is spot on.
FDA Chief Makary says rare diseases are huge a priority, but $CAPR received a CRL full of errors (blamed on AI) The study data had a p value of 0.0066 - statistically significant. Please give the DMD Boys hope.
@DrMakaryFDA@US_FDA@SecKennedy If rare diseases are huge a priority, how come $CAPR received a CRL full of errors ???(blamed on AI) The study data had a p value of 0.0066 - statistically significant.
A patient story from @Elijahjstacy below
https://t.co/pAwrEyzCuA
Time for FDA to walk the talk
@DrMakaryFDA@US_FDA@SecKennedy If rare diseases are huge a priority, how come $CAPR received a CRL full of errors ???(blamed on AI) The study data had a p value of 0.0066 - statistically significant.
A patient story from @Elijahjstacy below
https://t.co/pAwrEyzCuA
Time for FDA to walk the talk
As the founder and leader of a DMD nonprofit, I meet and speak to patients and parents on a daily basis. They want deramiocel and it gives a lot of hope to patients, me included, starting to face cardiac decline.
Every single day, weekends included, I have been working on helping get this approved. I am going up to bat for the DMD community and myself. I am now publicly calling on, as a patient, for other PATIENT advocacy groups and patients to start to speak publicly about their need and desire to get access to deramiocel.
What I want to make clear is this isnโt just another little drug that will make small improvements, but a drug that can potentially rewrite the prognosis of DMD by adding decades to oneโs life. #1 cause of death in DMD is cardiomyopathy and youโre potentially addressing that issue completely in one go. As a patient, itโd be very nice to remove/greatly reduce the biggest cause of death from my things to worry about.
So letโs come together, DMD community, and make this happen. I have big plans to help get this through, but Iโll take more than just me. Letโs go win!
@Elijahjstacy Please send Capricor CRL Response letter to the Senator you have met with and the others you can.
The responses in this letter paint a clear picture for why deramiocel should have been approved and FDA had no statistical argument to issue the CRL.
https://t.co/nmzN9Hqdvh
$CAPR Response to CRL
There are no surprises in this letter. FDA staff must be embarrassed that they have to deal with Prasad's flawed judgment. The case made by $CAPR in this document is rock solid.
I want to emphasize that I find the responses related to statistics to be particularly strong and to be consistent with the highest standards of statistical analysis.
https://t.co/swGVbsNsgW