Title: | Correction of Biochemical Abnormalities and Improved Muscle Function in a Phase I/II Clinical Trial of Clenbuterol in Pompe Disease |
Journal : | Molecular therapy : the journal of the American Society of Gene Therapy, 26, 9 |
Authors: | Koeberl DD, Author ; Koeberl DD ; Case LE ; Smith EC ; Walters C ; Han SO ; Li Y ; Chen W ; Hornik CP ; Huffman KM ; Kraus WE ; Thurberg BL ; Corcoran DL ; Bali D ; Bursac N ; Kishnani PS |
Material Type: | Article |
Publication Date: | 2018 |
Size: | p 2304 |
Languages: | English |
Keywords: | acid alpha-glucosidase;enzyme replacement therapy;glycogen storage disease; |
Pubmed / DOI : | DOI : 10.1016/j.ymthe.2018.06.023 / Pubmed : 30025991 |
N° Profil MNM : | 2018072 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/30025991 |
See also : |