Skip to main content
Full Schedule
Full Schedule
Browse by Title
Browse by Community
Browse by Network
Posters
Browse by Number
Browse by Presenter
Browse by Title
Speakers
Presidential Trainees
Awardees
Career Center
Partner Directory
Livestream Technical Support
Toggle navigation
Login
Search
Home
+
Full Schedule
Full Schedule
Browse by Title
Browse by Community
Browse by Network
+
Posters
Browse by Number
Browse by Presenter
Browse by Title
Speakers
Presidential Trainees
Awardees
Career Center
Partner Directory
Livestream Technical Support
Tweets by ASCPT 2023 Annual Meeting
Rare Diseases (RD)
Home
Rare Diseases (RD)
Rare Diseases (RD)
Type here to filter the list
EP-023 - POPULATION PHARMACOKINETIC MODELLING TO CONFIRM WEIGHT-BASED BANDED DOSING AND EXPOSURE-RESPONSE EFFICACY ANALYSES TO SUPPORT TROFINETIDE TREATMENT IN RETT SYNDROME.
Favorite
EP-024 - POPULATION PHARMACOKINETIC MODELLING TO PREDICT TROFINETIDE EXPOSURE AND EXPOSURE-RESPONSE SAFETY ANALYSES IN GIRLS WITH RETT SYNDROME AGED 2–4 YEARS.
Favorite
PI-081 - ELEXACAFTOR/TEZACAFTOR/IVACAFTOR (ETI) -INDUCED REACTIVE OXYGEN SPECIES (ROS) PRODUCTION IN HEPG2 CELLS: A POTENTIAL MECHANISM OF DRUG-INDUCED LIVER INJURY (DILI)
Favorite
PI-082 - PLASMA METHYLMALONIC ACID CONCENTRATION IS POSITIVELY ASSOCIATED WITH METABOLIC DECOMPENSATION EVENTS IN PATIENTS WITH METHYLMALONIC ACIDEMIA.
Favorite
PII-104 - INDIVIDUAL DOSE OPTIMIZATION FOR SOMATROGON BY MODELING AND SHINY INTERFACE.
Favorite
PII-105 - INVESTIGATION OF THE BIOTRANSFORMATION OF TRIHEXYPHENIDYL AND APPLICATION TO DYSTONIC CEREBRAL PALSY.
Favorite
PII-106 - PLASMA 3-HYDROXYPROPIONIC ACID CONCENTRATION IS POSITIVELY ASSOCIATED WITH METABOLIC DECOMPENSATION EVENTS IN PATIENTS WITH PROPIONIC ACIDEMIA.
Favorite
Back to Top
