Scholars@Duke publication: Phase 1 Study of Gene Therapy in Late-Onset Pompe Disease: Analyses of Safety and Secondary Endpoints

Phase 1 Study of Gene Therapy in Late-Onset Pompe Disease: Analyses of Safety and Secondary Endpoints

Publication , Conference

Koeberl, D; Smith, EC; Case, L; Hopkins, S; Childers, MK; Dearmey, S; Walters, C; Han, S-O; Spears, TG; Hornik, CP; Bali, D; Kishnani, PS

Published in: MOLECULAR THERAPY

2020

MOLECULAR THERAPY

1525-0024

1525-0016

2020

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Chicago

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NLM

Koeberl, D., Smith, E. C., Case, L., Hopkins, S., Childers, M. K., Dearmey, S., … Kishnani, P. S. (2020). Phase 1 Study of Gene Therapy in Late-Onset Pompe Disease: Analyses of Safety and Secondary Endpoints. In MOLECULAR THERAPY (Vol. 28, pp. 224–224).

Koeberl, Dwight, Edward C. Smith, Laura Case, Sam Hopkins, Martin K. Childers, Stephanie Dearmey, Crista Walters, et al. “Phase 1 Study of Gene Therapy in Late-Onset Pompe Disease: Analyses of Safety and Secondary Endpoints.” In MOLECULAR THERAPY, 28:224–224, 2020.

Koeberl D, Smith EC, Case L, Hopkins S, Childers MK, Dearmey S, et al. Phase 1 Study of Gene Therapy in Late-Onset Pompe Disease: Analyses of Safety and Secondary Endpoints. In: MOLECULAR THERAPY. 2020. p. 224–224.

Koeberl D, Smith EC, Case L, Hopkins S, Childers MK, Dearmey S, Walters C, Han S-O, Spears TG, Hornik CP, Bali D, Kishnani PS. Phase 1 Study of Gene Therapy in Late-Onset Pompe Disease: Analyses of Safety and Secondary Endpoints. MOLECULAR THERAPY. 2020. p. 224–224.

MOLECULAR THERAPY

1525-0024

1525-0016

2020

224 / 224