@Pierrette Design and outcome measures of LAVENDER, a phase 3 study of #trofinetide
for #Rett syndrome

https://www.sciencedirect.com/science/article/pii/S1551714422000301?via%3Dihub

FDA OKs First Drug for #Rett Syndrome

📌 The FDA has approved #trofinetide oral solution as the first treatment of Rett syndrome in adults and children aged 2 years and older.

📌Rett syndrome is a rare, genetic neurodevelopmental disorder that affects about 6000 to 9000 people in the United States, mostly females.

📌Symptoms typically present between 6 and 18 months of age, with patients experiencing a rapid decline with loss of fine motor and communication skills.

https://www.medscape.com/viewarticle/989649?src=rss&icd=login_success_email_match_norm

@paulhellyer
Thanks for sharing, wouldn't have seen this otherwise!
This is HUGE. Well done to that magnificent woman and her team
#DameMargaretBrimble #RettSyndrome #Trofinetide