Evaluation of cardiac function in patients with <scp>SMA</scp> after treatment with onasemnogene abeparvovec
Subclinical myocardial dysfunction after infusion of onasemnogene abeparvovec was suggested by careful investigation of cardiac function. Cardiac toxicity may correlate with liver impairment following gene replacement therapy. Long-term studies that allow for a higher number of patients and more extended observation periods should be conducted to confirm the cardiac toxicity of onasemnogene abeparvovec.
Authors
- Keiji Goishi
- Ayumi Mizukami
- Reiko Arakawa (ORCID: https://orcid.org/0000-0002-0910-8910)
- Junko Yamanaka (ORCID: https://orcid.org/0000-0001-6752-3664)
- Satoshi Takasago
- Hideko Uryu
- Masao Kaneshige
- Katsuyoshi Shimozawa (ORCID: https://orcid.org/0000-0002-9285-4649)
- Akihisa Horigome (ORCID: https://orcid.org/0000-0003-4275-2158)
- Hiroyuki Shichino (ORCID: https://orcid.org/0000-0002-0915-2110)
Institutions
- Nihon University (JP)
- National Center for Global Health and Medicine (JP)
Publication Details
- Journal
- Pediatrics International
- Published
- 2025-01-01
- DOI
- https://doi.org/10.1111/ped.70019
- Primary Topic
- Neurogenetic and Muscular Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00