Sustained clinical benefit following systemic gene replacement therapy in Duchenne muscular dystrophy

Muscle Nerve. 2024 Jan;69(1):4-6. doi: 10.1002/mus.28000. Epub 2023 Nov 16.

Authors

Hugh J McMillan^{1

2}, Hanns Lochmüller^{1

2

3}

Affiliations

¹ Children's Hospital of Eastern Ontario, University of Ottawa, Ottawa, Ontario, Canada.
² Brain and Mind Research Institute, University of Ottawa, Ottawa, Ontario, Canada.
³ The Ottawa Hospital Research Institute, University of Ottawa, Ottawa, Ontario, Canada.

PMID: 37969074
DOI: 10.1002/mus.28000

No abstract available

Publication types

Editorial

MeSH terms

Dystrophin / genetics
Exons
Genetic Therapy
Humans
Muscular Dystrophy, Duchenne* / genetics
Muscular Dystrophy, Duchenne* / therapy

Substances

Dystrophin

Grants and funding