I am confident he’s being serious re AdComs. Thats good for FDA and it’s great for patients. Put drugs on “trial” for everyone to see.

Yoshinobu Yamamoto, 98mph Fastball and 93mph Splitter, Overlay.

I follow FDA news and policy, but did not know about the problem of AdComms until today. Thank you to this author for pointing out even more broken bits at FDA that need a cast. Please fix this. @FDA_KyleD, @SecKennedy & @POTUS. @houmanhemmati @peter_mantas @mike98572986 @PeterPitts @SenRonJohnson @SenRickScott @RepHarshbarger washingtonexaminer.com/op-ed…

$qure $clpt While we all wait for the update on the type b meeting this article shows the complete saga over the last 12-24 months. This amt-130 situation could be made into a 2 hour movie or a Netflix documentary with the insane drama that has occurred. What is even more crazy about this saga is that you have all this drama and this is only the publicly available side of the story. Can you imagine one day when you get the behind the scenes views of Uniqure, Tabrizi/Sung, internal fda members, patients in the trial, it will get much more crazy. Don’t think I saw this article before, it was on May 5th, one of the better recaps of the entire Uniqure situation that I have seen. Hopefully for the HD community we get an update soon that is considered fair by them. Some of the interesting passages in the article below. Really shows the devastation that Prasad has occurred, history likely will look very bad on Prasad, mainly due to his intense desire to try to bury this program. “The cost is measured in patient-years - in the people for whom every additional regulatory month is a measurable, irreversible loss of cognitive and motor function, and in the people who will reach the end of their statistical disease horizons before the trial the FDA is now requiring is even completed. Those people exist. They are named. They have families. They have read the same data the FDA read. They have read the same agreement the FDA signed and unsigned. They are asking what changed. They deserve an answer.” “What the FDA has not addressed publicly is that uniQure used a sham control in approximately 10 patients earlier in the AMT-130 program and then stopped using it, precisely because of ethical concerns about asking patients with a fatal disease to undergo a procedure with no therapeutic benefit. According to a clinical analysis published by chemistry blogger and longtime industry observer Derek Lowe at Science.org, roughly 10% of those original sham-arm patients developed a blood clot during the procedure, which involved 10 to 12 hours of general anesthesia.” “Bioethics professor Holly Fernandez Lynch of the University of Pennsylvania, asked about the FDA's practice of attacking uniQure on background calls with reporters, said: "The fact that you are sharing information with the press without standing behind it either suggests that you know you should not be talking to the press or that you don't really stand behind your opinion. Why are we debating this in the court of public opinion? The stakes are too high." “Several other companies have publicly requested adcomm meetings, and at least three have said the FDA initially told them to prepare for a meeting and then retracted that guidance. On the same March 5 background call where the senior FDA official attacked uniQure and singled out Woodcock by name, the official also dismissed the broader pool of outside scientists who serve on FDA advisory panels, saying they "do not do their homework" and may be motivated by financial conflicts of interest. The agency's position is that an advisory committee is unnecessary. The agency's former Acting Commissioner, multiple senators, the rare disease community, and at least three other companies that have requested adcomms believe an advisory committee is precisely what is required.” “The agency has granted four expedited designations to AMT-130 specifically: Fast Track in 2019, Orphan Drug, RMAT in 2024, and Breakthrough Therapy in 2025. Each designation is supposed to mean the FDA recognizes both the unmet need and the substantial preliminary evidence of effectiveness. Each was granted on the same data the agency now describes as unable to demonstrate "any therapeutic benefit." davemanuel.com/2026/05/05/fd…

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

Shohei Ohtani's production is even BETTER than you might think so far this season! 🤯 BK digs in on how incredible the Dodgers star is playing.