ProjektportalPharmacometric evaluation of treatment effects in rare diseases
Terrmin
VT22
Beskrivning
In the era of individualized therapies, some diseases stand out as rare and are subsequently less explored. Recently, studies have intensified to individualize treatments for rare diseases. “N of one experiment” is the term that has been coined to signify the scarce number of individuals that are enrolled in those trials. Regulatory agencies have also highlighted the importance of those investigations and recommended new methods for their assessment; for example, in a recent response to a newly discovered anti-sense oligonucleotide (ASO) therapy specifically designed for the treatment of an ultra-rare disease, the head of the FDA has stated that the evaluation of disease trends before and after treatment will be the primary method of assessing treatment efficacy in rare diseases.
We would like to welcome a master’s student who will explore the pharmacometric evaluation of treatment effects for a chosen rare disease. The student will work conjointly with Mohamed Gewily (PhD student) and under the supervision of Prof. Mats Karlsson from the pharmacometrics group. The student will use Non-linear mixed effect models (NLME) to assess the treatment efficacy, and at the end of their project, the student will be asked to write a thesis to illustrate the findings of their work.
The supervision will be in English. The project is suitable for a student who has taken the elective courses: Models for Biological Systems and Clinical PKPD.
Huvudområde
Farmaceutisk vetenskap
Ämne
Farmakokinetik
Typ
Laborativ studie
Företag
Uppsala University
Ort/Plats
Uppsala
Handledarens namn
Mohamed Gewily
Handledarens e-post
mohamed.gewily@farmaci.uu.se
Institution
Program
Kurs
Omfattning/hp
30hp
Hur många studenter kan antagas för detta projekt?
1