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development and plasticity [Figure 3]. The inclusion of candidate therapies that target both
mitochondrial dysfunction and ameliorate oxidative stress may have some potential benefits for patients if
included in the treatment regime of patients from the early stages of the disease.
CONCLUSION
Many different LSDs present with psychosis and neurological dysfunction, including patients with AM.
These clinical manifestations are still poorly understood within AM disease presentation, and AM requires
further elucidation into its pathophysiology, mechanisms, and long-term outcomes in relation to its
neurologic and psychiatric presentation. There have been some clinical trials into the effectiveness of HSCT
and ERT in AM patients, although the latter has no reported evidence of any neurologic improvement in
these patients due to the inability of ERT to cross the BBB. HSCT appears to be the better option for AM
treatment, particularly for the amelioration of neurocognitive symptoms and prevention of further
neurodegenerative decline, despite the lack of clinical data to show improvement and/or prevention of
psychosis and neurocognitive dysfunction in adult AM patients.
DECLARATIONS
Acknowledgments
The authors would like to thank all the patients with Alpha Mannosidosis.
Authors’ contributions
Study concept and design: Dewsbury MR, Hargreaves IP, Stepien KM
Critical review of the first draft: Morgan HM
Literature search and wrote the first draft of the manuscript writing: Dewsbury MR
Manuscript editing: Dewsbury MR, Hargreaves IP, Morgan HM, Stepien KM
Availability of data and materials
Not applicable.
Financial support and sponsorship
None.
Conflicts of interest
Stepien KM is a CI for Alpha Mannosidosis Registry. The other authors declared that there are no conflicts
of interest.
Ethical approval and consent to participate
Not applicable.
Consent for publication
Not applicable.
Copyright
© The Author(s) 2024.
REFERENCES
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