Clinical development innovation in rare diseases: overcoming barriers to successful delivery of a randomised clinical trial in alkaptonuria—a mini-review
Abstract Alkaptonuria is a rare inherited disorder for which there was no disease-modifying treatment. In order to develop a successful approved therapy of AKU multiple barriers had to be overcome. These included activities before the conduct of the study including deciding on the drug therapy, the...
Αποθηκεύτηκε σε:
| Κύριοι συγγραφείς: | , |
|---|---|
| Μορφή: | Artigo |
| Γλώσσα: | Inglês |
| Έκδοση: |
BMC
2023-01-01
|
| Σειρά: | Orphanet Journal of Rare Diseases |
| Θέματα: | |
| Διαθέσιμο Online: | https://doi.org/10.1186/s13023-022-02606-0 |
| Ετικέτες: |
Δεν υπάρχουν, Καταχωρήστε ετικέτα πρώτοι!
|