| nr |
titel |
auteur |
tijdschrift |
jaar |
jaarg. |
afl. |
pagina('s) |
type |
| 1 |
Achieving big with small: quantitative clinical pharmacology tools for drug development in pediatric rare diseases
|
Ahmed, Mariam A.
|
|
|
50 |
6 |
p. 429-444
|
artikel
|
| 2 |
Challenges, approaches and enablers: effectively triangulating towards dose selection in pediatric rare diseases
|
Durairaj, Chandrasekar
|
|
|
50 |
6 |
p. 445-459
|
artikel
|
| 3 |
Empirical bayes approach for dynamic bayesian borrowing for clinical trials in rare diseases
|
Sebastien, Bernard
|
|
|
50 |
6 |
p. 495-499
|
artikel
|
| 4 |
External control arms for rare diseases: building a body of supporting evidence
|
Khachatryan, Artak
|
|
|
50 |
6 |
p. 501-506
|
artikel
|
| 5 |
Population pharmacokinetic modeling and dosing simulation of avalglucosidase alfa for selecting alternative dosing regimen in pediatric patients with late-onset pompe disease
|
Tiraboschi, Gilles
|
|
|
50 |
6 |
p. 461-474
|
artikel
|
| 6 |
Rare oncology therapeutics: review of clinical pharmacology package of drug approvals (2019–2023) by US FDA, best practices and recommendations
|
Mitra, Amitava
|
|
|
50 |
6 |
p. 475-493
|
artikel
|
| 7 |
Special issue: Model-informed drug development in rare diseases: connecting the dots in an information rich ecosystem
|
Krishna, Rajesh
|
|
|
50 |
6 |
p. 425-427
|
artikel
|
| 8 |
The future of rare disease drug development: the rare disease cures accelerator data analytics platform (RDCA-DAP)
|
Barrett, Jeffrey S.
|
|
|
50 |
6 |
p. 507-519
|
artikel
|
Tijdschrift beschrijving