What's new and what's next for gene therapy in Pompe disease?
Roger AL, Sethi R, Huston ML, Scarrow E, Bao-Dai J, Lai E, Biswas DD, El Haddad L, Strickland LM, Kishnani PS, ElMallah MK.
Roger AL, et al. Among authors: strickland lm.
Expert Opin Biol Ther. 2022 Sep;22(9):1117-1135. doi: 10.1080/14712598.2022.2067476. Epub 2022 Apr 27.
Expert Opin Biol Ther. 2022.
PMID: 35428407
Free PMC article.