Today we delivered a petition to the FDA that garnered nearly 20,000 signatures FDA, we expect a response and definitive action for Barth syndrome patients and families! https://t.co/L5A8CMB2Ot
Happy #PKUday2023 to the whole #PKU community! Our commitment to supporting patients and families living with #Phenylketonuria is increasing day by day!
We are always by your side! 😊❤️
https://t.co/hjv7qbzFlZ
#PKUDAY
#InternationalPkuday 2023 is today!
The birthdays of both Dr Horst Bickel (pioneer of #PKU treatment) and Robert Guthrie (newborn screening inventor) were both on 28th June!
Groundbreaking work helping thousands and thousands of people live lives with less/no brain damage
👌👌👌
At #STATBreakthrough, Dr. Bob Lustig calls GLP-1 weight loss drugs “band-aid” for problem of obesity, not a fix. Points out people lose fat AND muscle.
“This is what starvation looks like.”
Did you get a notice that says your personal info was exposed in a data breach? Did you lose your wallet? Or learn that an online account was hacked? Depending on what information was lost or exposed, there are steps you can take to help protect yourself: https://t.co/ei8O0WnuGo
I believe that every American has a right to the peace of mind that comes with knowing they have access to affordable, quality health care.
The Inflation Reduction Act is a historic investment in that belief.
Pharmalot.. Pharmalittle.. Good Morning: Global demand for monkeypox vaccine will be hard to meet; Amgen dispute with IRS heats up & More news.. https://t.co/wbPNt1feEI #pharma#monkeypox#vaccines#taxes#cancer $AMGN $BMY $MRK $SNY $BVNRY $AZN $SRPT
I spent the past few months reporting this story out, learning about the infuriating gap between clinical success and commercial failure for SCID gene therapy.
Check out Jakob's story here, which is playing out in many rare diseases beyond 'bubble boy'
https://t.co/wTFtE7JbNR
The body of evidence for the protective benefit of masks, updated with multiple new studies, for community and health care settings
https://t.co/1hsoxKYaNX @AnnalsofIM