Title: | Applying the win ratio method in clinical trials of orphan drugs: an analysis of data from the COMET trial of avalglucosidase alfa in patients with late-onset Pompe disease. |
Journal : | Orphanet journal of rare diseases, 19, 1 |
Authors: | Boentert M ; Berger KI ; Diaz-Manera J ; Dimachkie MM ; Hamed A ; Riou França L ; Thibault N ; Shukla P ; Ishak J ; Caro JJ |
Material Type: | Article |
Publication Date: | 12/01/2024 |
Size: | p 14 |
Languages: | English |
Keywords : | enzymotherapy ; late-onset Pompe disease ; outcome measure ; Pompe disease ; statistics ; therapeutic efficiency |
Keywords: | Avalglucosidase alfa ; Clinical trial ; Enzyme replacement therapy ; Late-onset Pompe disease ; Win ratio |
Lien associé : | Texte complet disponible sur PubMed Central (PMC) |
Pubmed / DOI : | Pubmed : 38216959 / DOI : 10.1186/s13023-023-02974-1 |
N° Profil MNM : | 2024012 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/38216959 |