Mother of boys and other animals. Writer. Ideas-seeker. Love books + real talk. Red Sox Nation lifer. Duchenne warrior: https://teapaw.org/t.co/OOrB4PQqJj
Joined January 2011
- Tweets1.7K
- Following672
- Followers294
- Likes21.2K
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-woods-ca…
1
4
229
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
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
healthaffairs.org/content/fo…
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.
4
6
289
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.
9
193
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 teapaw.org/i/broadcasts/1kvJpMEyz…
Jennifer Handt retweeted
Happening Today at 11am: Advancing Rare Disease Detection and Treatment teapaw.org/i/broadcasts/1kvJpMEyz…
$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.
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: bit.ly/3Kkhw22
$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
Hochul Sets Up Death Panel for Muscular Dystrophy Kids dlvr.it/TPCD5y
Jennifer Handt retweeted
Families of terminally ill kids deserve action, not excuses. NY spends billions in tax dollars, yet parents are told their children’s lives “cost too much”? Every level of government must step up. Families need support, and $SRPT must ensure these kids get the medicine they need.
$SRPT “New York’s leadership brags about equity, inclusion, and compassion -- yet its health department just told families of terminally ill boys that their children’s lives cost too much.” This writer calls it what it is: a death panel. @NYSDOHMEDICAID
In states like New York, it’s not a conspiracy theory — it’s official policy.
americanthinker.com/blog/202…
$SRPT Another single-center study (CHOP). "All patients showed improvements in motor outcomes."
$SRPT
Philadelphia Children’s Hospital
hemostasistoday.com/science/…
Jennifer Handt retweeted
#Duchenne News: Today, Satellos announced that the first patient has been dosed in LT-001, an open-label, long-term follow-up study of SAT-3247 in adult males with Duchenne muscular dystrophy. Full details here: tinyurl.com/satellos-fpd
#MuscleRegeneration $MSCL $MSCLF
@ParentProjectMD "Our results...indicat[e]strong interest in gene therapy as a treatment for DMD, even given a hypothetical scenario in which the benefit is described as non-curative, time limited, and with a treatment-related risk of death." $SRPT
sciencedirect.com/science/ar…
$SRPT Real-world data from commercially dosed patients at a single center.
"There are 7,000 rare diseases. We can treat about 700 or 800 of them. You know, take that money that you're putting behind another me too GLP-1 or another obesity drug, which is seemingly a market that is already well-satisfied, and go do something worthwhile in society." 👏
We also heard from GSK CEO Emma Walmsley, Biogen CEO Christopher Viehbacher.
Maraganore on the rise of China in Biotech: "We need to look at this as a Sputnik moment as a country, if we want to maintain our leadership in the US as the leading biomedical industry country. We need to change. We need to accelerate our abilities to get evidence faster. We need to think about the cost of our research and development activities and how to do it cheaper. We have to be competitive with what China is doing."
Emma Walmsley on vaccines: ""We are cautious about the US right now, but in the rest of the world, vaccines are doing very well. Tragically, when vaccination rates drop, infection rates rise. People die unnecessarily. And then vaccination rates come back up."
Viehbacher on GLP-1s: "There are 7,000 rare diseases. We can treat about 700 or 800 of them. You know, take that money that you're putting behind another me too GLP-1 or another obesity drug, which is seemingly a market that is already well-satisfied, and go do something worthwhile in society and invest in something that's more [of a] breakthrough."
#STATSummit
"There's no fiscal line item for lost time."
This Post is from an account that no longer exists. Learn more
$SRPT My son is a data point on this slide. Can confirm these results stand up IRL.
"At 2 years, Part 1-treated patients showed statistically significant and clinically meaningful functional improvement vs. the matched EC cohort."
investorrelations.sarepta.co…
