Phase 3 Randomized Trial Results of DTX401 AAV Gene Therapy for the Treatment of GSDIa.

Mitchell JJ, Abdenur JE, de Boer F, Boyer M, Breilyn MS, Couce ML, De Leon DD, Derks TG, El-Gharbawy A, Haijer-Schreuder AB, Loechner K, Longo N, Lund AM, Olmos MAM, McCandless SE, de Oliveira BM, Mount M, Muschol N, Pytlak K, Radhakrishnan K, Riba-Wolman R, Rodriguez-Buritica DF, La Rosa A, Rossi A, Saavedra H, Santer R, Shayota B, Smit GPA, Moura De Souza CF, van der Klauw MM, Weinstein DA, Wolfsdorf JI, Blake A, Grimm AA, Mitragotri D, Rahman S, Turner-Bowker DM, Collis R

Open source

DOI: 10.1002/jimd.70241
Published: 2026 Sep
Container: Journal of inherited metabolic disease
Publisher: Not recorded
Open access: yes

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Cite this work

BibTeX

@article{allodium:10.1002/jimd.70241,
  title = {Phase 3 Randomized Trial Results of DTX401 AAV Gene Therapy for the Treatment of GSDIa.},
  author = {Mitchell JJ and Abdenur JE and de Boer F and Boyer M and Breilyn MS and Couce ML and De Leon DD and Derks TG and El-Gharbawy A and Haijer-Schreuder AB and Loechner K and Longo N and Lund AM and Olmos MAM and McCandless SE and de Oliveira BM and Mount M and Muschol N and Pytlak K and Radhakrishnan K and Riba-Wolman R and Rodriguez-Buritica DF and La Rosa A and Rossi A and Saavedra H and Santer R and Shayota B and Smit GPA and Moura De Souza CF and van der Klauw MM and Weinstein DA and Wolfsdorf JI and Blake A and Grimm AA and Mitragotri D and Rahman S and Turner-Bowker DM and Collis R},
  year = {2026},
  journal = {Journal of inherited metabolic disease},
  doi = {10.1002/jimd.70241},
  url = {https://doi.org/10.1002/jimd.70241}
}

RIS

TY  - JOUR
TI  - Phase 3 Randomized Trial Results of DTX401 AAV Gene Therapy for the Treatment of GSDIa.
AU  - Mitchell JJ
AU  - Abdenur JE
AU  - de Boer F
AU  - Boyer M
AU  - Breilyn MS
AU  - Couce ML
AU  - De Leon DD
AU  - Derks TG
AU  - El-Gharbawy A
AU  - Haijer-Schreuder AB
AU  - Loechner K
AU  - Longo N
AU  - Lund AM
AU  - Olmos MAM
AU  - McCandless SE
AU  - de Oliveira BM
AU  - Mount M
AU  - Muschol N
AU  - Pytlak K
AU  - Radhakrishnan K
AU  - Riba-Wolman R
AU  - Rodriguez-Buritica DF
AU  - La Rosa A
AU  - Rossi A
AU  - Saavedra H
AU  - Santer R
AU  - Shayota B
AU  - Smit GPA
AU  - Moura De Souza CF
AU  - van der Klauw MM
AU  - Weinstein DA
AU  - Wolfsdorf JI
AU  - Blake A
AU  - Grimm AA
AU  - Mitragotri D
AU  - Rahman S
AU  - Turner-Bowker DM
AU  - Collis R
PY  - 2026
JO  - Journal of inherited metabolic disease
DO  - 10.1002/jimd.70241
UR  - https://doi.org/10.1002/jimd.70241
ER  -

APA

JJ, M., JE, A., F, D. B., M, B., MS, B., ML, C., DD, D. L., TG, D., A, E., AB, H., K, L., N, L., AM, L., MAM, O., SE, M., BM, D. O., M, M., N, M., K, P., K, R., R, R., DF, R., A, L. R., A, R., H, S., R, S., B, S., GPA, S., CF, M. D. S., MM, V. D. K., DA, W., JI, W., A, B., AA, G., D, M., S, R., DM, T., & R, C. (2026). Phase 3 Randomized Trial Results of DTX401 AAV Gene Therapy for the Treatment of GSDIa.. Journal of inherited metabolic disease. https://doi.org/10.1002/jimd.70241

Source records

pubmed · retrieved 2026-09-25T01:40:00.011Z