All about Cystic Fibrosis. Always refer to your team for specialist advice. Oh and we don't always agree with RTs.
Joined February 2012
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cf/Aware retweeted
Excited to offer participation in GRAMPUS-CF to patients with cystic fibrosis worldwide. Just click on the link to join the study! grampus-cf.org @cftrust @CF_Foundation @alloonsl @CFAware
cf/Aware retweeted
$VRTX on pace to lose $15B in market value today.
$VRTX - That was a bad quarter. Every product missed. Lower end of guidance raised -- who cares? Lots of handwaving around Journavx access but insignificant revenue. Casgevy is a money loser.
The stock is near an all-time high and trades at 26x, Vertex needs to do better.
The company seems more dependent than ever on its pipeline for future growth.
Analysts mostly shrug.
cf/Aware retweeted
Today the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for our fifth cystic fibrosis (CF) medicine. This is an important step towards our goal of treating all people living with CF.
cf/Aware retweeted
Today the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for the label expansion of our fourth cystic fibrosis (CF) medicine to include an expanded population with certain mutations. This marks an important step towards our goal of treating all people with CF.
Mycobacterium Abscessus Infections and Cystic Fibrosis | Nature Research Intelligence nature.com/research-intellig…
Cystic Fibrosis and Fungal Infections | Nature Research Intelligence nature.com/research-intellig…
Cystic Fibrosis and Bone Health | Nature Research Intelligence nature.com/research-intellig…
Do intravenous antibiotics improve pulmonary exacerbations (flare-ups) in people with cystic fibrosis? | Cochrane cochrane.org/CD009730/CENTRA…
Body composition changes and clinical outcomes in pediatric cystic fibrosis during 24 months of lumacaftor ivacaftor therapy based on real-world data | Scientific Reports nature.com/articles/s41598-0…
cf/Aware retweeted
🚨 New in The Lancet Respiratory Medicine
Phase 3 data on vanzacaftor–tezacaftor–deutivacaftor in #cysticfibrosis
Read open access here:
Patients aged 6–11 years tinyurl.com/446ju86t
Patients aged 12 years and older tinyurl.com/5y453b56
cf/Aware retweeted
GRAMPUS-CF aims to understand gut symptoms in people with cystic fibrosis (CF)
so that we can develop treatments. If you are a person with CF, you can help! Visit
our website for participant information, online consent and to download the app.
grampus-cf.org/ Thank you!
NICE Board says new method allowing greater weight to be given to severe diseases is working | NICE nice.org.uk/news/articles/ni…
Boy, 3, has combination of CFTR mutations, no CF symptoms: Report cysticfibrosisnewstoday.com/…
NICE approves three cystic fibrosis drugs for NHS use - The Pharmaceutical Journal pharmaceutical-journal.com/a…
cf/Aware retweeted
UK #CysticFibrosis community! Tell us about your thoughts on inhaled meds & interacting with your care providers post-Kaftrio in this anonymous 5-10 min research survey. Your voice matters! Please share! [shef.qualtrics.com/jfe/form/…] #CF
cf/Aware retweeted
Trikafta revolutionized treatment for Cystic Fibrosis, reaching >90% of patients variants.
But what about the remaining untreatable 10%?
We studied several poorly responsive, FDA-unapproved variants and paved a path forward for drug development.
biorxiv.org/content/10.1101/… 1/7
CF patients eat high-fat diet despite guidelines: Review cysticfibrosisnewstoday.com/…
