Vamorolone Safety, Pharmacokinetics, and Exploratory Efficacy in Duchenne Muscular Dystrophy: A Phase II, Nonrandomized, Multiple-Dose Study in 2-<4-Year-Old Boys.

Mah JK, Gonorazky HD, Nigro E, Lochmüller H, Alemán A, Yaworski A, Oskoui M, Sbrocchi AM, Selby KA, de Vera A, Mathur R, Gresko E, Linden A, Dutreix C, Hoffman EP

Open source

DOI
10.1212/wnl.0000000000218066
Published
2026 Jun 9
Container
Neurology
Publisher
Not recorded
Open access
yes

Credibility signals

limited evidence Score 45/100 under policy 1.0.0. This is a metadata assessment, not a judgment of the paper's conclusions.

Show all credibility signals

Cite this work

BibTeX

@article{allodium:10.1212/wnl.0000000000218066,
  title = {Vamorolone Safety, Pharmacokinetics, and Exploratory Efficacy in Duchenne Muscular Dystrophy: A Phase II, Nonrandomized, Multiple-Dose Study in 2-<4-Year-Old Boys.},
  author = {Mah JK and Gonorazky HD and Nigro E and Lochmüller H and Alemán A and Yaworski A and Oskoui M and Sbrocchi AM and Selby KA and de Vera A and Mathur R and Gresko E and Linden A and Dutreix C and Hoffman EP},
  year = {2026},
  journal = {Neurology},
  doi = {10.1212/wnl.0000000000218066},
  url = {https://doi.org/10.1212/wnl.0000000000218066}
}

RIS

TY  - JOUR
TI  - Vamorolone Safety, Pharmacokinetics, and Exploratory Efficacy in Duchenne Muscular Dystrophy: A Phase II, Nonrandomized, Multiple-Dose Study in 2-<4-Year-Old Boys.
AU  - Mah JK
AU  - Gonorazky HD
AU  - Nigro E
AU  - Lochmüller H
AU  - Alemán A
AU  - Yaworski A
AU  - Oskoui M
AU  - Sbrocchi AM
AU  - Selby KA
AU  - de Vera A
AU  - Mathur R
AU  - Gresko E
AU  - Linden A
AU  - Dutreix C
AU  - Hoffman EP
PY  - 2026
JO  - Neurology
DO  - 10.1212/wnl.0000000000218066
UR  - https://doi.org/10.1212/wnl.0000000000218066
ER  - 

APA

JK, M., HD, G., E, N., H, L., A, A., A, Y., M, O., AM, S., KA, S., A, D. V., R, M., E, G., A, L., C, D., & EP, H. (2026). Vamorolone Safety, Pharmacokinetics, and Exploratory Efficacy in Duchenne Muscular Dystrophy: A Phase II, Nonrandomized, Multiple-Dose Study in 2-<4-Year-Old Boys.. Neurology. https://doi.org/10.1212/wnl.0000000000218066

Source records