Without FDA flexibility for rare diseases our daughter’s future is at risk Explore FDA flexibility in medical care as a mother shares her daughter's inspiring journey through health challenges.

For instance:
#Galactosemia and #Govorestat. We are desperate!

Or #PDCD. The kid in this article should have been dead by the age of four, got a promising new medication, is now six, and will now be denied the medication? I am furious on behalf of them. 😡
rarerevolutionmagazine.com/without-fda-...

2/
Det er rundt 25 i Norge med tilstanden.
SORD deficiency finner jeg ikke på norsk i det hele tatt, de ble jeg klar over fordi de kanskje får lov å få #govorestat, som også skal være effektivt på galaktosemi. (Lang historie)

Weekly Roundup - PHARMA - 16 December 2024 🔊AstraZeneca's Imfinzi Wins FDA Approval for Limited-Stage Small Cell Lung Cancer AstraZeneca's Imfinzi offers a new post-chemotherapy immunotherapy. The ADRIATIC trial showed survival benefits, makin...

Weekly Roundup - PHARMA - 16 December 2024

www.linkedin.com/pulse/weekly...

kstrategyand.com/pharma-news

#AstraZeneca #Imfinzi #Govorestat #Ozempic #Delgocitinib #SaudiArabia #GenScript #Tobevibart #Elebsiran #NeuroSense #PrimeC #Humanteconomy #Humantec #KStrategyand

Applied Therapeutics Crashes After FDA Rejection of Rare Disease Drug After extending its review period to evaluate additional submissions, the FDA ultimately denied Applied Therapeutics’ govorestat for galactosemia, citing “deficiencies” with the application. The biote...

Well that was a kick in the teeth for investors and patients alike. On the bright side, the stock is at an amazing price now. Hope #APLT can get this resolved. #Govorestat

www.biospace.com/fda/applied-...

Wait for galactosemia therapy goes on after FDA shuns Applied Therapeutics’ govorestat - Pharmaceutical Technology The US Food and Drug Administration has declined to approve Applied Therapeutics’ govorestat for classic galactosemia.

I am totally gutted.
😭😭😭
#Galactosemia patients still have to wait for #Govorestat/ #AT-007

www.pharmaceutical-technology.com/news/galacto...

Govorestat Faces FDA Rejection, Leaving Galactosemia Patients Without Hope The FDA's denial of Govorestat, the sole potential treatment for Classic Galactosemia, leaves patients and families in despair. Advocacy continues for their reconsideration.

Govorestat Faces FDA Rejection, Leaving Galactosemia Patients Without Hope #USA #Albany #Galactosemia_Foundation #Govorestat #Classic_Galactosemia

We can no longer trust that other countries mirroring NIH will lead to best practice if this man gets the chair.
I am so tired of fighting for "my" diseases and disorders, two of which are just about to get their first medications out.🤯
#Galactosemia
#Govorestat
#WolframSyndrome
#ALS
#AMX0035