The goal of this observational study is to learn the effects of the drug velmanase alfa (Lamzede®) in the bodies of children under the age of 3 with Alpha-Mannosidosis.
The main questions it aims to answer are:
* study the effect of velmanase alfa on a marker of the disease called GlcNAc(Man)2 after one year of therapy * explore how the child's body reacts to velmanase alfa during the therapy The parents or legal guardians of participants will be asked to provide the results of analyses performed in the routine clinical setting related to the participant's general health and the administration of velmanase alfa.
Additional data will be extracted from other observational sponsored studies/registries, compassionate use programs, investigator-initiated studies (IIS), and published case reports (presented in the literature) if existing.
Subjects with the provision of informed consent from their legal guardians (LAR)
Have a confirmed diagnosis of alpha mannosidosis
Have initiated treatment with velmanase alfa between birth to at least six weeks before turning 3 years of age
None
Paediatric patients with a confirmed diagnosis of alpha mannosidosis via MAN2B1 genetic testing and/or alpha-mannosidase activity in leukocytes or fibroblasts \< 10% of normal activity, with data for at least one pre- and one post-Lamzede treatment sample obtained when \< 3 YOA, after at least 6 weeks of treatment (primary analyses).
Other
Other
Velmanase Alfa
DrugLamzede® (velmanase alfa, henceforth referred to as Lamzede) is a recombinant human lysosomal alpha-mannosidase product developed as an intravenous enzyme replacement therapy (ERT) for the treatment of alpha-mannosidosis.
Pharmacodynamic Response to velmanase alfa
Change (absolute and relative) of GlcNAc(Man)2 level from pre-velmanase alfa treatment baseline in blood
Development of anti drug antibody to velmanase alfa
Assessment of the impact of anti drug antibody on pharmacodinamic
Treatment-emergent adverse events
Number of undesirable events not present prior to medical treatment, or an already present event that worsens either in intensity or frequency following the treatment
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