About Alpha-Mannosidosis
Alpha-mannosidosis is a lysosomal storage disorder caused by deficiency of lysosomal alpha-mannosidase (MAN2B1), which is required to break down mannose-rich oligosaccharides. Accumulation of these glycoprotein fragments in cells throughout the body causes intellectual disability, recurrent infections due to immune dysfunction, hearing loss, and skeletal abnormalities. Velmanase alfa (Lamzede) is an approved enzyme replacement therapy for non-neurological manifestations.
Common Clinical Features
Clinical Trial Eligibility Tips
What to know before applying to Alpha-Mannosidosis trials.
Velmanase alfa (Lamzede) is approved — trials may focus on CNS outcomes, higher-dose regimens, or next-generation approaches
Urinary oligosaccharide excretion pattern is the diagnostic biomarker required for trial documentation
Neuropsychological testing battery results are key eligibility and outcome measures
Hematopoietic stem cell transplantation has been used in some patients — transplant status affects eligibility for ERT or gene therapy trials
Patient Resources
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