Jennifer Handt

Mother of boys and other animals. Writer. Ideas-seeker. Love books + real talk. Red Sox Nation lifer. Duchenne warrior:

[Jennifer Handt @JenniferHandt]

@jasongay this is the correct take. https://t.co/B0CkuTjItS

[Jennifer Handt @JenniferHandt]

It's not easy being Tiger? That's the conclusion? He has almost killed people with his car, multiple times. He needs to figure his stuff out like every other grown adult on the planet.

https://t.co/EYfcv8qNH2

[Jennifer Handt @JenniferHandt]

@edcollins23 @Elijahjstacy Thanks 🙏🏼

[Jennifer Handt @JenniferHandt]

@FischNancy @DrMakaryFDA @SecKennedy @SenRonJohnson @SenRickScott @POTUS @dirod923 This is incredible.

[Jennifer Handt @JenniferHandt]

@yachmod it's absolutely shocking. Bring back the ombudsman!

[Jennifer Handt @JenniferHandt]

Nothing gets my Irish up like a bean counter telling me my son's therapy doesn't work. Robert Kaplan doesn't know Duchenne, he doesn't get rare disease drug development and he certainly doesn't know my son. $SRPT

[Health Affairs @Health_Affairs]

Accelerated approval can serve patients well—but only when surrogate endpoints are reliable and clearly predict outcomes that matter to patients. | Forefront

Robert M Kaplan | @StanfordCERC

https://t.co/fKb3NOacvz

[Jennifer Handt @JenniferHandt]

@Health_Affairs https://t.co/EwdSENO7A7

[Jennifer Handt @JenniferHandt]

Hey @health_affairs here's an idea for your suggestion box. If you're going to cover this first-of-its-kind drug that is literally changing the course of this horrid disease maybe make sure coverage includes the latest data which is conveniently absent in here.

[Jennifer Handt @JenniferHandt]

@Health_Affairs More revolutionary: include...I dunno...the patient's point of view? I'd be glad to tell your readers how my son GAINED two points on his NSAA in the past six months at a time when he should be declining. But by all means tell me whether gene therapy works or not.

[Jennifer Handt @JenniferHandt]

This action will get approved and anticipated treatments to children faster, will end the dx odyssey for families and will level the field going forward to align us all toward combination therapies and new & better treatment options. It's a monumental step in the right direction.

[Jennifer Handt @JenniferHandt]

As an advocate for newborn screening at the federal and state levels, I commend @HHSGov for taking the long-awaited step of adding #Duchenne to the Recommended Uniform Screening Panel to identify the #DMD diagnosis in newborns.

[HHS @HHSGov]

Happening Today at 11am: Advancing Rare Disease Detection and Treatment https://t.co/d2Jp5DhSaO

[HHS @HHSGov]

Happening Today at 11am: Advancing Rare Disease Detection and Treatment https://t.co/d2Jp5DhSaO

[Jennifer Handt @JenniferHandt]

$SRPT Good news for the DMD community & our shared understanding of treatment safety. I want to honor the patients & families who enroll in this study. Science comes with risk, and it takes courage to accept that risk in the pursuit of something better than your bad genetic luck.

[Sarepta Therapeutics @Sarepta]

We just announced that the U.S. FDA approved to begin dosing in Cohort 8 of the ENDEAVOR study to evaluate the use of an enhanced immunosuppressive regimen as part of treatment with our gene therapy for non-ambulatory patients. Read the press release: https://t.co/8h4x7YX4dZ

[Jennifer Handt @JenniferHandt]

@yachmod They'll attack Sarepta for underdisclosing, then attack them for overdisclosing.

[Jennifer Handt @JenniferHandt]

$SRPT this is not hyperbole. This is inappropriate wielding of power to decide who gets to live or die. And let's remember how many of the DURB members have ties to industry. @NYSDOHMEDICAID @HealthNYGov

[RealClearHealth @RealClearHealth]

Hochul Sets Up Death Panel for Muscular Dystrophy Kids https://t.co/3ugXtDDd7E

[Jennifer Handt @JenniferHandt]

@TequilaVernetti Of course they don't—it doesn't suit the narrative.

[Jennifer Handt @JenniferHandt]

@TequilaVernetti They talk like they've never sat across a doctor's desk and heard that their child is terminal and there is nothing they can offer but corticosteroids. And they act like we can't see improvement with our lying eyes.

[Jennifer Handt @JenniferHandt]

@commonsenseplay I appreciate your nuanced look at it. It's an intellectual exercise few care to make. And you know what happens when people with power (the pen, policy, investors) come at these companies? Families like mine lose.

[Jennifer Handt @JenniferHandt]

@commonsenseplay Researchers have been at this disease since the gene was discovered in 1986. In 5 years as an advocate post-dx, I have not seen a single set of DMD data I have not had to squint at. This monster is shifty and its victims are as heterogenous as it gets. This ish is hard.