Marty Makary left behind an FDA families learned not to trust



With so much bad news in the world, it is worth pausing for one encouraging development: Marty Makary finally resigned as commissioner of the Food and Drug Administration last week.

Makary’s tenure at the FDA was marred by internal scandals, forced resignations, dreadful morale, and record staff turnover. More important, he actively sandbagged President Trump’s push to expand clinical trials for rare diseases through the aptly named “right-to-try” framework.

Trump’s next appointee should restore the spirit of right to try and make safe, effective treatments available to children as quickly as possible.

The idea behind right to try is straightforward. Patients with rare conditions, especially those for whom conventional medicine has failed, should have the freedom to pursue experimental treatments that have not yet received full FDA approval. Families fighting the clock have little left to lose. Government should not stand between them and a potentially lifesaving breakthrough.

Makary did.

Members of the MPS community sent more than 10 letters asking Makary for a meeting. They got a form letter in return. Sen. Ron Johnson (R-Wis.) later announced an investigation into the FDA’s denials. Makary’s agency responded by claiming approvals were already “at their peak.” The Wall Street Journal took notice of the FDA’s foot-dragging last year, yet the agency kept rejecting relevant rare-disease treatments in early 2026, including RGX-121 and drugs from Biohaven and Saol Therapeutics.

That stonewalling forced families to escalate.

In March, more than 100 mothers and other advocates staged a mock funeral outside FDA offices. Dressed in black and carrying a real coffin, they sought to draw attention to a group of rare metabolic disorders known as mucopolysaccharidoses. These disorders can show up as mild symptoms such as depression or hyperactivity, or as devastating conditions such as heart disease and skeletal abnormalities.

Many MPS disorders still have no approved treatments, even though they can severely diminish children’s quality of life or kill them outright. The FDA’s regulatory process serves a legitimate purpose. But when a bureaucracy grows so rigid, self-protective, and arrogant that it blocks desperately ill children from access to promising therapies, it stops functioning as a safeguard and starts functioning as a death sentence.

Mark Dant of the Ryan Foundation told Newsweek that some of these drugs were denied because of the FDA’s institutional “dislike” of the accelerated-approval pathway. “For decades we waited for science to find our tomorrows,” he said. “Now it has, and bureaucrats within the agency we pay for are keeping those treatments from our children. We know they are there. … We just cannot reach them.”

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Valerie Plesch/Bloomberg/Getty Images

Makary’s resignation will not undo the damage. But it does create an opening. We may not yet know what the FDA’s next leadership will look like, but Trump’s appointee should restore the spirit of right to try and make safe, effective treatments available to children as quickly as possible.

Across the world, in the nation of Georgia, parents have staged a protest lasting more than 500 consecutive days, maintaining a round-the-clock presence outside the main government building in Tbilisi. They are willing to risk everything to give their children the best chance at life. Americans should not have to camp outside federal offices for 500 days to get their government to listen.

The new FDA leadership must explain denials of right-to-try clinical trials with enough specificity that sponsors and families understand what evidence could change the decision. Patient and caregiver testimony should shape decisions early, not get folded in at the end as a token gesture. And Congress must demand transparency without turning each drug review into a partisan circus.

Children’s lives are not bargaining chips. The FDA exists to serve the public, not to protect its own bureaucracy from embarrassment. If Makary’s departure opens the door to that truth, families battling ultra-rare diseases may finally have reason to hope.

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The FDA seems to care more about celebrities than sick Americans



Last month, while many veterans celebrated Joe Rogan and President Donald Trump’s support for psychedelic drugs, those in the Huntington’s disease community like me faced another disappointment. UniQure, a company with a promising treatment, may be abandoning the U.S. market because of bureaucratic roadblocks.

I’m not the president or the world’s most popular podcaster. What I am is a daughter who has tested positive for the Huntington’s disease gene and will one day exhibit the same symptoms of this disease that ate away at my father’s personality and his mind until he took his own life.

The FDA’s answer always seems to be the same when it comes to rare disease treatments: Wait, wait, and then wait some more.

I have advocated for the Huntington’s disease community, both in my father’s memory and with the hope that my future will be different from his. The outlook is dim for those like me unless the Food & Drug Administration allows access to treatments like AMT-130, which UniQure is now advancing first in the U.K. after the FDA’s unreasonable demands pushed the United States down the priority list.

Those demands are disastrous for Huntington’s disease patients. Launching a placebo trial under the FDA’s proposed new criteria would require non-therapeutic injections into the brains of study patients — hardly aligning with medical ethics.

Even without the basic inhumanity of this type of trial, Huntington's patients simply cannot afford the years it would take to complete it. We are living on a much shorter timeline, defined by a merciless disease that is both progressive and fatal.

I’m glad that veterans are getting the attention they deserve and that they have the support of influencers like Rogan. But it raises an important question: Why should it take a celebrity and the president to push the FDA to follow basic common sense and medical best practices?

For years, the rare-disease community has done everything we were told would make a difference. We organized, advocated, and pushed for change with whatever strength we had, often while managing devastating diagnoses and worsening symptoms.

Parents of children with Duchenne muscular dystrophy and Sanfilippo syndrome, to name but two groups, have advocated while watching their children decline.

The FDA’s answer always seems to be the same when it comes to rare disease treatments: Wait, wait, and then wait some more. That means we’re running down hours on a clock that ticks ominously louder with every passing month. We don’t have time for years of unnecessary testing.

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Tom Merton/Getty Images

Rogan’s intervention shows that the system can move quickly when it wants to — certainly the president will listen when voices with direct access amplify a cause. Now we need to see that same urgency applied to treatments for rare diseases.

Families like mine are not looking for special treatment. We are only asking for the choice to take the risk of trying new medicines when all the old options have failed. After all, we know the future that awaits us.

President Trump already made the right move with the Right to Try Act, which gives terminally ill patients a pathway to access potentially lifesaving or life-extending treatments. It is critical that he push FDA officials to commit to the same right-to-try principles he championed in his first term.

Scientists are making incredible strides in treating rare diseases. But that innovation only matters if patients are allowed to use treatments already developed. Adults like me, and kids with terminal rare diseases whose parents approve, are absolutely willing to accept any risk that comes with trying a new therapy.

Until someone steps up to bat for people like me, our only alternative is the certaintyof an illness that will slowly, relentlessly ruin our lives and then snuff them out.

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Chinese fraudster convicted for ripping off Americans with bogus COVID tests



Jia Bei Zhu, the Chinese national linked both to the secret California biolab discovered in 2022 and the biolab discovered earlier this year in Las Vegas, is finally reaping the whirlwind for his crimes on American soil.

The Justice Department announced on Wednesday that a jury has convicted Zhu on one count of conspiracy to commit wire fraud, eight counts of substantive wire fraud, two counts of distributing adulterated and misbranded medical devices, and one count of making a false statement to the Food and Drug Administration.

'He flouted the lawful authority of the FDA and deliberately deceived the public.'

While he now faces the possibility of decades in prison, Zhu has evaded justice for at least a decade.

The British Columbia Supreme Court determined in 2016 that Zhu and several companies he controlled had conspired to steal confidential technology from a Colorado-based company, partly with the intention to sell said technology in China.

Facing over $270 million in damages and prison time, the IP-stealing fraudster fled Canada in 2015, then entered the United States — "unlawfully," according to the DOJ — where he continued to rip off Americans.

The jury in the Chinaman's latest trial was presented with evidence that, once stateside, Zhu founded a company in California with his romantic partner, Zhaoyan Wang, called Universal Meditech Inc.

With the help of employees hired through the Fresno County Economic Development Corporation who "would not ask any questions," Zhu, Wang, and others at UMI conspired from August 2020 through March 2023 to import faulty COVID tests from China — the origin of the disease — then sell those faulty tests to Americans based on numerous false representations, prosecutors claimed.

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They falsely claimed that the tests were authorized by the Food and Drug Administration, were made in the U.S., were made in connection with a certified medical lab, and worked.

Zhu managed to rake in nearly $4 million selling over 1 million malfunctioning COVID tests.

According to the DOJ, Wang was charged in connection with the scheme but evaded arrest by fleeing to China.

Zhu's scheme first came to light when one of his victims filed a civil lawsuit in 2022, prompting an inspection of UMI's Fresno facility — an inspection which the DOJ said not only demonstrated UMI's inability to properly manufacture COVID tests but proved the company to be nothing more than "an unsanitary warehouse that was far below established quality standards for facilities that house medical devices."

Once again, Zhu attempted to escape justice, moving UMI from Fresno to Reedley and changing its name to Prestige Biotech Inc.

When the FDA began investigating him, Zhu lied about his immigration status and identity, claiming to be Qiang "David" He. Zhu also said he knew nothing about UMI or PBI.

The discovery of Zhu's Reedley biolab in early 2023 ushered in the end of the Chinaman's years-long grift.

Months after a code enforcement officer noticed a hose sticking out of a supposedly vacant warehouse in the heart of Reedley — a clear violation of the municipality's building code — local officials executed a search warrant on March 16, 2023.

Inside, they found lab equipment, trace narcotics, roughly 1,000 mice that were genetically engineered to mimic the human immune system, and faulty medical devices subject to an FDA health embargo along with "blood, tissue, and other bodily fluid samples and serums; and thousands of vials of unlabeled fluids and suspected biological material," according to a congressional report.

Zhu was arrested on Oct. 19, 2023.

While in custody, police raided another one of the Chinese national's properties, this time a Las Vegas residence managed by an Israeli national currently in the U.S. on an E-2 visa. The FBI said more lab equipment was discovered at the scene, including a "bio-safety hood, a bio-safety sticker, a centrifuge, multiple refrigerators, red-brown unknown liquids in gallon-sized containers, and refrigerated vials with unknown liquids."

Eric Grant, U.S. attorney for the Eastern District of California, said that the verdict in Zhu's case "holds the defendant accountable for actions that exploited a public health crisis for his own gain. He flouted the lawful authority of the FDA and deliberately deceived the public by repackaging low-quality, foreign-made test kits at a time when accuracy and reliability were critical."

Grant added, "This conduct, tied to the unlawful operations uncovered at the Reedley laboratory, put lives at risk."

The House Select Committee on the Chinese Communist Party has confirmed that Zhu is a Chinese citizen associated with communist regime-linked companies as well as with Chinese military-civil fusion entities.

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