Efficacy and safety of avalglucosidase alfa in Japanese patients with late-onset and infantile-onset Pompe diseases: A case series from clinical trials
Although the number of patients was small, avalglucosidase alfa provides an efficacy and safety profile in Japanese patients representative of the overall populations from key global clinical trials.
Authors
- Masato Ikeda (ORCID: https://orcid.org/0000-0003-3583-5975)
- Madoka Mori‐Yoshimura (ORCID: https://orcid.org/0000-0003-4312-9114)
- Satoko Kumada (ORCID: https://orcid.org/0000-0002-9687-7151)
- Hirotaka Ohki
- Hirofumi Komaki (ORCID: https://orcid.org/0000-0002-0659-1417)
- Hideaki Mashimo
- Kenji Inoue
- Takashi Kiyono
- Akihiro Arimori
Institutions
- Chiba University (JP)
- National Center of Neurology and Psychiatry (JP)
- Tokyo Metropolitan Neurological Hospital (JP)
- Chiba Hospital (JP)
- Sanofi (Japan) (JP)
- Tokyo Metropolitan Children's Medical Center (JP)
Publication Details
- Journal
- Molecular Genetics and Metabolism Reports
- Published
- 2024-12-27
- DOI
- https://doi.org/10.1016/j.ymgmr.2024.101163
- Citations
- 1
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.33
Funders
- Sanofi
- Sanofi K.K.