$capr I cannot share this impactful video enough. Currently listening to Linda speak today just reassures how powerful Deramiocel is for these young boys. Keep sharing with the world as the OP says so their voices are heard.
This is Kai and he’s a teenager with Duchenne Muscular Dystrophy, a genetic disease that kills most boys before 25. His life was improved when he joined a clinical trial for a promising drug called “Deramiocel” from Capricor Therapeutics.
Then that drug’s review process was put on pause by the FDA until the company refiled with the best data ever in treating DMD. Kids had 54% better function at the end of a clinical trial called HOPE-3. Thank goodness, it's finally under review again. Since 2025, FDA reviewers have rejected 23 rare disease drugs and some believe this was driven by Vinay, but others suggest it's the near-sighted FDA reviewers. Trump finally pushed Vinay out for his inability to do the job the American people demand. So there is hope that treatments for rare diseases may see some light.
Moms of dying kids don't care about the deep state’s "process." They care if their child sees Christmas. Acting U.S. FDA Commissioner Kyle Diamantas said he wants to reopen approvals for these rare disease drugs. Here’s hoping he’s serious.
And moves fast.
#CapricorPartner
This is Kai and he’s a teenager with Duchenne Muscular Dystrophy, a genetic disease that kills most boys before 25. His life was improved when he joined a clinical trial for a promising drug called “Deramiocel” from Capricor Therapeutics.
Then that drug’s review process was put on pause by the FDA until the company refiled with the best data ever in treating DMD. Kids had 54% better function at the end of a clinical trial called HOPE-3. Thank goodness, it's finally under review again. Since 2025, FDA reviewers have rejected 23 rare disease drugs and some believe this was driven by Vinay, but others suggest it's the near-sighted FDA reviewers. Trump finally pushed Vinay out for his inability to do the job the American people demand. So there is hope that treatments for rare diseases may see some light.
Moms of dying kids don't care about the deep state’s "process." They care if their child sees Christmas. Acting U.S. FDA Commissioner Kyle Diamantas said he wants to reopen approvals for these rare disease drugs. Here’s hoping he’s serious.
And moves fast.
#CapricorPartner
@NSPharmaInc@nipponshinyaku@Capricor The boys are still waiting. The data is still the best DMD data ever generated. The law, as it stands in the Third Circuit today, points toward remand. $capr
$capr
@NSPharmaInc@nipponshinyaku
NS’s “Notice of Removal” tells you plainly how they see the world strategy and priorities, and on optics it is not a good look for them. Getting in the way of DMD patients’ ability to access Deramiocel. We are watching! #DMD
@NSPharmaInc@nipponshinyaku NS is essentially asking Neals to treat this case as if it were an FAA/Convention enforcement action when it plainly is not. $capr
I think their delay tactics have finally hit a wall and @capricor next response will solidify that. DMD community is watching & we don’t have time
@JoelKatz@RippleXDev 14 years ago was the inception of XRP but if you include the years leading up to the the team finally creating/launching it, how many years in total is that of xrp in the making ?