Jason Mast

Five people sitting on the summit stage

Our first session of the day: A Milestone for Gene Editing

@jasonmast.bsky.social talks with Kyle and Nicole Muldoon, Rebecca Ahrens-Nicklas, and Kiran Musunuru about how CRISPR was used to correct a misspelling in KJ Muldoon genes when he was just 6 months old.

#STATSummit

CRISPR is used in landmark treatment to correct genetic misspelling of a single patient In a world first, scientists used CRISPR to fix a baby's unique genetic mutation. The report offers hope for personalized treatments of rare diseases.

If you want to understand why he is a celebrity, read this piece by @jasonmast.bsky.social

www.statnews.com/2025/05/15/c...

Parents of children with rare diseases ask: How long until our CRISPR miracle? How to better manage expectations around gene editing drugs for families of children with rare diseases.

Parents of children with rare diseases ask: How long until our CRISPR miracle?

www.statnews.com/2025/10/09/c...

HHS abruptly cancels CDC meeting with RFK Jr., anti-vaccine activist as scrutiny mounts HHS abruptly canceled a CDC meeting between RFK Jr. and anti-vaccine activist David Geier just hours before they were set to gather.

HHS abruptly cancels CDC meeting with RFK Jr., anti-vaccine activist as scrutiny mounts www.statnews.com/2025/09/17/r... via @statnews.com

A hopeful thread! Some of my favs:
HIV treatment is better by leaps and bounds, and now 2x year injections can prevent people from contracting it! www.npr.org/sections/goa...

CRISPR has cured, CURED, sickle cell anemia in patients for at least a year! www.scientificamerican.com/article/fda-...

Piercing the hype around Patrick Soon-Shiong's 'Bioshield' cancer treatment Why trial results presented earlier this week do not support the claim made by billionaire physician Patrick Soon-Shiong.

Patrick Soon-Shiong has (still) not cured cancer
www.statnews.com/2025/09/11/s...

Child dies after receiving new experimental gene therapy The first child to receive a new experimental gene therapy for a rare neurological disorder died a few days after dosing, the company sponsoring the trial said.

The first child treated with a new gene therapy designed to safely reach deep into the brain died just days after dosing

If tied to treatment, this would be the 5th death linked to gene therapy in the past year. Since 2019, there have been at least 13

www.statnews.com/2025/09/10/c...

Tracking RFK Jr.’s promises to remake health in America STAT introduces a goals and promises tracker for Robert F. Kennedy Jr.'s Make America Healthy Again movement — successes, incompletes, and fails.

Love this tracker from @isabellacueto.bsky.social and @jaspar.bsky.social chronicling all of RFK's promises on chronic disease and public and how much he's delivered — or not — on them
www.statnews.com/2025/09/10/r...

Big shakeups to the childhood vaccination schedule could be nearing Big shakeups may be coming to the childhood vaccination schedule. Here's a look at some of the shots that could be subject to shifting U.S. recommendations.

Big shakeups to the childhood vaccination schedule could be nearing
via @elaineywchen.bsky.social
www.statnews.com/2025/09/03/c...

Wild to see a thread about me. I think the broader topic (as Jason points out) is what does the future of preventive medicines look like for at risk gene carriers? I also hope this gives people some hope to those dealing with devastating and (previously) unactionable inherited genetic diseases.

Trump Budget Office Is Withholding H.I.V. Funds That Congress Appropriated

Trump proposes to rescind AIDS funds. Congress rejects the proposal. So Trump and his henchman Russ Vought just refuse to spend the money and try to hide that decision from Congress and the public. www.nytimes.com/2025/08/21/h...

Trump executive order seeks to centralize control of grantmaking under political appointees A sweeping new executive order seeks to transform how the federal government awards billions of dollars in research grants

Trump executive order seeks to centralize control of grantmaking under political appointees www.statnews.com/2025/08/08/t... via @statnews.com

Rare disease patients caught in Trump crackdown on foreign grant awards Rare disease research has been caught up in a Trump administration crackdown on NIH grants being used to support international collaborations

Rare disease patients have been caught in Trump crackdown on foreign grant awards

An important story from @mmolteni.bsky.social
www.statnews.com/2025/08/06/n...

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Did a drug prevent this man’s ALS? Jeff Vierstra was likely doomed by his DNA. A radical experiment gave him a chance to rewrite his fate — before ALS symptoms ever began.

Prevention — not treatment — for neurodegenerative diseases.

Jeff appears to have been the first test case. Five years later, the early results are in:
www.statnews.com/2025/07/28/a...

Did a drug prevent this man’s ALS? Jeff Vierstra was likely doomed by his DNA. A radical experiment gave him a chance to rewrite his fate — before ALS symptoms ever began.

It was also a bold first experiment of an approach that may one day be applied to thousands of other people living with what can feel like ticking time bombs wired into their DNA: Genes for Huntington's disease, familial forms of Alzheimer's and ALS, Prion disease, among others

Did a drug prevent this man’s ALS? Jeff Vierstra was likely doomed by his DNA. A radical experiment gave him a chance to rewrite his fate — before ALS symptoms ever began.

But the results were "abnormal." Given his gene, the doctor feared he was seeing early, silent signs of ALS. He wanted to put Jeff on the drug, despite the serious risks it might entail. The drug, largely untested, could do significant damage.

It was an effort to save Jeff's life and prevent ALS

Did a drug prevent this man’s ALS? Jeff Vierstra was likely doomed by his DNA. A radical experiment gave him a chance to rewrite his fate — before ALS symptoms ever began.

Then, in 2020, two of his sisters began showing symptoms. Thanks to a chance-meeting, they were each given early access to an experimental drug designed to suppress the toxic gene.

Jeff then got his own nerve-function test. The results worried his neurologist. He didn't have ALS — not yet at least

Did a drug prevent this man’s ALS? Jeff Vierstra was likely doomed by his DNA. A radical experiment gave him a chance to rewrite his fate — before ALS symptoms ever began.

A 🧵: Last week, I wrote about Jeff Vierstra, who carries the gene for a devastating, rare form of ALS. The disease killed his mom and all three of her siblings, along with relatives dating back to the 1800s.

Then, in 2020, two of his sisters began showing symptoms.

Did a drug prevent this man’s ALS? For over an hour, Jeff Vierstra lay still in Columbia University’s ALS clinic, as a doctor poked him ankle-to-throat with an electric needle. Sometimes,

He didn't have ALS. At least, not yet. But clearly some of his nerve cells had died. Would an experimental drug prevent him from getting it?

Great piece from @jasonmast.bsky.social

www.statnews.com/2025/07/28/a...

NIH is shrinking the number of research projects it funds due to a new Trump policy NIH plans to shrink the share of grant applications it will award for the remainder of the fiscal year, in some cases by more than half.

Last month, I wrote about a new Trump policy requiring NIH to fund multiyear awards upfront, and concerns that it would shrink the number of new grants NIH could support. Those fears are now being realized. From me, @aniloza.bsky.social, & @angusrohan.bsky.social :

www.statnews.com/2025/07/29/n...

In an event celebrating the 35th anniversary of the passage of the Americans with Disabilities Act, HHS Secretary Kennedy spent 8ish minutes talking about how his family started the Special Olympics, his disabled aunt Rosemary, and democracy. He then posed for a picture.

He did not mention the ADA.

Thanks Alyssa!

For many Duchenne families, halt to gene therapy treatments is heartbreak upon heartbreak As gene therapy maker Sarepta Therapeutics tangles with FDA over its Duchenne treatment, patients and their families are caught in the middle.

The dust-up between Sarepta Therapeutics and the FDA has rightly generated all kinds of headlines lately. But this one, from @jasonmast.bsky.social, is about those most affected by it all: Duchenne patients and their families.

www.statnews.com/2025/07/24/d...

For many Duchenne families, halt to gene therapy treatments is heartbreak upon heartbreak As gene therapy maker Sarepta Therapeutics tangles with FDA over its Duchenne treatment, patients and their families are caught in the middle.

@jasonmast.bsky.social hears the confusion, fear, and heartbreak for Duchenne families across the country. His story at @statnews.com

www.statnews.com/2025/07/24/d...

How Trans Journalists Can Stay Safe and Well While Changing Their Bylines - The Open Notebook Changing a name is a very personal decision. Unfortunately for trans journalists, it’s a decision that has to occur in public. Changing a byline can mitigate the abuse and harassment that trans journa...

Are you a trans journalist trying to change your byline? You should check out this guide I have been writing for the last few months for @theopennotebook.bsky.social, which is full of tips/tricks from me and many other trans journalists.

www.theopennotebook.com/2025/07/22/h...

We're a journalist-founded website chronicling the #LongCOVID crisis. Unlike others, we continue to report on the impact of the ongoing COVID-19 pandemic. No denial. No minimizing. No gaslighting.

Here's our editorial team + writers who've contributed to our publication. go.bsky.app/8zDRx6Y

In the aftermath of Hurricane Helene, when there was no power, internet, cell service, or running water for weeks.. Blue Ridge Public Radio was our region's only source for critical, life-saving information. Everyone would gather around crank/battery powered radios 2x daily for updates. #npr

"As the NIH begins to reinstate these grants, it is creating a surprising map of winners and losers at odds with the Trump administration’s politics." great story by @aniloza.bsky.social www.statnews.com/2025/07/03/n...