P404: Alpha-mannosidosis: A case report on diagnostic challenges, enzyme replacement therapy, and ongoing hematopoietic cell transplantation
In May 2024, we initiated enzyme replacement therapy (ERT) with Velmanase alfa (Lamzede), approved by the FDA on February 16, 2023. The patient received weekly infusions and tolerated them well without side effects.
Authors
- Subhadra Ramanathan
- Hisham Abdel‐Azim (ORCID: https://orcid.org/0000-0003-0880-8701)
- Hua Wang (ORCID: https://orcid.org/0000-0002-2605-5697)
- Sajad Khazai
Institutions
- Loma Linda University (US)
Publication Details
- Journal
- Genetics in Medicine Open
- Published
- 2025-01-01
- DOI
- https://doi.org/10.1016/j.gimo.2025.103271
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00