Top Tweets for #Govorestat
@DrMakaryFDA You want proof a drug works?
Look at what happens when patients lose access to it.
We’re about to find out and we shouldn’t have to. For 6 years I’ve asked FDA for help.
#govorestat #CRL #galactosemia #citizenspetition #FIOA
P-values don’t work when the global patient population is the size of a kindergarten class.
Ultra-rare diseases need Bayesian tools not impossible standards. Drug abandonment in ultra rare is real. #govorestat #ultrarare #RareDisease
We were never set up for success at the FDA. 42 patients. 11 missing assessments. A drug denied—not because it failed, but because the system did. Ultra-rare disease deserves a new path forward. #Galactosemia #RareDisease #FDAReform #Advocacy #govorestat
💡Key events in the current lawsuit against #AppliedTherapeutics:
~ Throughout 2024, Applied Therapeutics made optimistic claims about #Govorestat for galactosemia.
~ Feb 15, 2024: Positive Phase III trial results announced, showing improved patient outcomes.
~ Nov 27, 2024: FDA issues Complete Response Letter citing deficiencies in Govorestat’s NDA.
~ The following day, $APLT dropped over 76%.
$APLT #APLT #AppliedTherapeutics
Check out Laura Jung's video describing FDA’s press release yesterday😭😭. #galactosemia #govorestat #raredisease #TikTok https://t.co/yJkMeRWtHN
I will be clicking refresh every 5 minutes waiting for our much needed approval #rare #raredisease #galactosemia #govorestat FDA Press Announcements
Source: U.S. Food and Drug Administration https://t.co/bWGGxN4noD
P has been on ACTIVE drug #govorestat for one month. First month no side effects. First positive; Constipation gone, for years we have done miralax daily and suppository weekly. Yesterday I put these meds away. Kids with Galactosemia have constipation so bad that they are at risk for megacolon. The once weekly crying and screaming followed by exhausted sleep is all gone. I don’t have to worry about scheduling life around this one symptom. #FDA @DrCaliff_FDA #raredisease #galactosemia
Had the worst dreams last night about my #raredisease child. Thought I lost her, it brought me back to her first few days of life where I almost did. She’s almost six but it stays with me. #galactosemia #PaveTheWayFDA #govorestat
The body makes more galactose than is found in milk 7.12mg/100g. It’s a ridiculous to think that a dairy free #diet alone can help #classicgalactosemia it’s a false safety net we have been told for too long. We need #govorestat which reduces galactitol by 40% Our own body make thousands more mgs. #pavethewayfda @FDACDERDirector @DrCaliff_FDA
Beslutningsforum blir trusselen for alle oss som lever nært en sjelden diagnose. I stedet for trygghet skaper de en ytterligere forsinkelse.
Håper de er snillere med #govorestat 😢
3-åringen er kortvokst. Medisinen som kan hjelpe ham, får han ikke i Norge
https://t.co/yK16xaFmZF
Question: “What’s your experience communicating with the FDA?”
Answer: A big sigh, a laugh, and eye rolls from everyone. 😤🤣🤣🙄🙄🙄
#journalist #patients #govorestat #raredisease #als #ms #fda #hillstaffer #shareholder #galactosemia #congress #senate #pavethewayfda #drugtrials #fda #covid #kpsc #sda #psi #cdc #bigpharma #painmeds #vaccine #kas #health #kannada #upsc #gk #vaccines #iso #ias #who #freedom #coronavirus #pfizer #pharmaceutical #lnp #kpsckannadiga #gmp #bidenharris #fdaapproved #usa
Kayla K. Parent-
“Today, we were informed that Kaden has been on placebo which was no surprise since we did not see the same rate of improvements in him as other friends during this journey. While #govorestat is not a cure, it doesn’t tackle every symptom of disease, it has shown to significantly increase cognitive abilities, increase activities of daily living skills, positive adaptive behavior changes, elimination of tremors, and has allowed those receiving medication the ability to bond and learn alongside their peers”
#pavethewayfda #thisisgalactosemia #galactosemia #raredisease #fda #change #petition @DrCaliff_FDA @FDAPatientinfo @Applied_Tx @RareDiseases
#FDA Please remember we are waiting for our treatment!!! Please place a priority review on our drug AT-007. #Govorestat demonstrated consistent long-term clinical outcomes benefit across a range of functional measures in the ACTION- #Galactosemia Kids trial, confirming prior #biomarker data @US_FDA @DrCaliff_FDA @RepCurtis80304
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