In case you missed it, this was a heartbreaking and insightful special. I hope you consider watching it & #WriteYourReps to support #ScienceResearch #SupportResearch #ScienceIsCool #Cures #RareDiseases #Autoimmune #CRISPR #Science #CNBCCures #Science #Healthcare #Migente
Reposting from @cnbc.com
Watched nearly entire #CNBCCures livestream. Was kind of shocked Santorum said as much against the FDA and this administration as he did. Really wish they didn’t let the politicians have that much of a platform here though. This event was about #RareDisease and it really took away from that.
One of the main sponsors of the event was GeneDX, who just happened to be the lab who confirmed my KRIT1 mutation for Familial CCM. Maybe one day we can find a treatment or cure.
#CNBCCures
CNBC CURES THE BIGGEST ENEMY CYSTIC FIBROSIS EVER HAD BOOMER ESIASON Former NFL Quarterback GUNNAR ESIASON RA Ventures
Well, the #CNBCCures livestream cut out in the middle of Boomer Esiason telling the story of his son’s diagnosis with cystic fibrosis.
Really wanted to hear from Gunnar. Hopefully @cnbc.com can get the video posted at some point.
Dr. Leonard Schleifer, Regeneron:
“We’re not going to make any money on this.”
“We try not to put commercial input in what we’re developing until the very, very end.”
Damn, this hurts and really cements what those living with a #RareDisease fear the most, even if there is a treatment.
#CNBCCures
Dr. Allyson Berent, Foundation for Angleman Syndrome Therapeutics: We know the risk of doing nothing, which is so much greater than the risk of doing something.
#CNBCCures
The FDA approved 20 drugs via the Accelerated Approval pathway in 2024—in 2025 the FDA only approved 9 drugs via accelerated approval said Gottlieb at the CNBC Cures summit focused on rare diseases #CNBCCures #medsky
The FDA's oncology drugs review staff is down to <60 from 100 employees said ex FDA Commissioner Scott Gottlieb ('17-'19) on panel at #CNBCCures conf moderated by Becky Quick.HIgh # of senior reviewers are gone & v difficult to replace. Will probably see a higher # of CRLs in '26 he said #medsky
Doug Ingram: We need to nurture innovation or we won’t have it in the future. I’m convinced of that.
#CNBCCures
Doug Ingram: We don’t think thoughtfully enough about safety and efficacy.
#CNBCCures
Doug Ingram, Sarepta: (talking about FDA) We need to do something different. Reduce requirements that don’t match current science. Balance risk of therapy vs. delay.
#CNBCCures
CNBC Cures THE UNCOMFORTABLE TRUTH ABOUT FUNDING PETER KOLCHINSKY RA Capital Management DR. WILL SHRANK Aradigm Health
Peter Kolchinsky: It is outrage and power of shame that drives where insurance money is being spent.
#CNBCCures
We are trying to help families who, like mine, are dealing with a rare disease. BECKY QUICK CNBC Anchor & mom of a child with a rare disease
Investing in rare diseases is not only a good investment, but the right thing to do for the millions suffering and challenged by rare disease. NICOLE BOICE Global Genes Founder
Patients are central to understanding rare diseases - they deserve a seat at the table. PAM GAVIN NORD CEO
I started by fighting for my family, and continue today by advocating for the people I meet and whose stories I share. JOHN CROWLEY Biotechnology Innovation Organization President & CEO
Some quotes being shown during a break in the #CNBCCures livestream. Picks back up at 340pm.
Fidji Simo from OpenAI talking about how ChatGPT saved a bad medical situation in a hospital setting with POTS seems like it’s selling the AI assistant part way too much.
We need to fix the healthcare system. Relying on AI as a fix for inadequate staffing isn’t the answer.
#CNBCCures
CNBC Cures REDEFINING WHAT'S POSSIBLE DR. ANNA GREKA Harvard Medical School DR. STAN CROOKE n-Lorem Foundation
Anyone who’s been to a medical or healthcare event online or in-person in the past, panels like these are the ones that make the event worth it.
Please, more medical professionals like these and way less political types if there is another #CNBCCures in the future.
Chris Viehbacher, CEO Biogen: “Once you have a treatment, the motivation to diagnose correctly goes up.”
Very true for #RareDisease. Many don’t bother with testing when there are no treatments or cures. Even with more known diseases like Alzheimer’s, very few get biomarker testing.
#CNBCCures
CNBC Cures Unlocking The Gridlock At The FDA Congressman Jake Auchincloss (D-MA) Dr. Scott Gottlieb Former FDA Commissioner Rick Santorum Former Senator (R-PA)
Didn’t really expect to hear from Rick Santorum today. Shockingly, he’s very critical of the FDA and this administration. #CNBCCures
“When rules slow innovation, patients pay the price.” #CNBCCures
The first ever #CNBCCures Summit starts in about 5 minutes. Hopefully this event is something helpful and inspiring for those living with rare diseases.
Registration is free for anyone that wants to follow along.
