Patients' and caregivers' maximum acceptable risk of death for non-curative gene therapy to treat Duchenne muscular dystrophy
Molecular Genetics & Genomic Medicine
March 2021
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Full Citation
Peay HL, Fischer R, Mange B, Paquin RS, Smith EC, Sadosky A, Russo L, Ricotti V, Rensch C, Morris C, Martin AS, Ganot A, Beaverson K, Mansfield C. Patients' and caregivers' maximum acceptable risk of death for non-curative gene therapy to treat Duchenne muscular dystrophy. Mol Genet Genomic Med. 2021 Mar 23:e1664. doi: 10.1002/mgg3.1664. Epub ahead of print. PMID: 33755338.
Tags
Journal Article Type
- Paper Resulting From PCORI-Funded Research Study (with results)
- Other Results
Journal Publication Name
- Molecular Genetics & Genomic Medicine
Literature Type
- PCORI Research