Jennifer Handt

@jasongay this is the correct take. wsj.com/sports/golf/ti…

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. golf.com/news/tiger-woo…

@edcollins23 @Elijahjstacy Thanks 🙏🏼

@JenniferHandt @Elijahjstacy s203.q4cdn.com/439234936/file…

This is very encouraging to me and explains some things imo 1) explains why the data visually looked worse than it actually is because small N makes the entire graph go down with one bad patient result 2) exercise seems to really matter imo because FVC you’re always breathing. I do not know what level of physical activity these patients are doing, but I know they’re constantly breathing, which stimulates the muscle. Dominate hand always appears to improve more

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

Posting this video everyday until @DrMakaryFDA reviews #ataluren using #realworldevidence This is what 9 years on #ataluren looks like, this is not the natural history for a 13 yr old with #duchenne @SecKennedy @SenRonJohnson @SenRickScott @POTUS #raredisease

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

@JenniferHandt How the heck is this even allowed to be published?

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 parentprojectmd.org/sarepta-shares…

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.

@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.

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.

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.

Happening Today at 11am: Advancing Rare Disease Detection and Treatment twitter.com/i/broadcasts/1…

$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.

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

$SRPT Elevidys, not much difference on black box warning for Zolgensma. Been on market for 6 years and has treated over 4,000 patients. Ask yourself if the hype on Elevidys’ warning has an agenda. zolgensma-hcp.com/clinical-trial…

$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

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

@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.

@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.

@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.

So much FUD circulating around $SRPT - HERE ARE THE FACTS! 1. Duchenne muscular dystrophy (DMD) patients have a median life expectancy of 22 years. Most boys die in their late teens / early 20s. 2. These boys don’t have unlimited therapeutic options. 3. ELEVIDYS (Sarepta's flagship gene therapy) is not available to everyone, it is contraindicated in patients with deletions in exon 8 and/or 9. 4. For those who aren’t eligible for ELEVIDYS (due to age, mutation, or antibodies), many are eligible for one of the 3 PMO exon-skipping therapies $SRPT markets which in those cases are literally their only option! 5. In their ESSENCE trial (which caused the huge drop after hours), the primary end point wasn’t met, but there were statistically significant functional benefits observed over 9 years, especially in patients who did not have COVID-era infusion disruptions. 6. PMOs deliver tangible clinical benefit, including: – +5.4 yrs median survival – 3–4 yr delay in loss of ambulation – ~30% reduction in ER visits and more (see chart below). Bottom line: these therapies meaningfully improve real-world outcomes in boys who otherwise face death in their early 20s. Any clinically meaningful benefit should remain an option. Families see benefit, that’s why they seek these therapies out. Don’t take away one of the only tools they have. This changes nothing for the $SRPT thesis, I expect a strong rebound as the FDA continues to keep these therapies accessible.