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Metazym

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Experimental recombinant enzyme
This article needs to be updated. Please help update this article to reflect recent events or newly available information. (November 2021)

Metazym is an experimental recombinant enzyme that was studied in patients with late infantile metachromatic leukodystrophy, but found to be ineffective under the conditions of that trial. A subsequent clinical trial is ongoing. The drug became a source of controversy when a family attempted to purchase the drug for their child before it was approved. Jonckheere, Kingma, Eyskens, Bordon, & Jansen (2023) highlight the shift towards the need for newborn screening for metachromatic leukodystrophy as it allows for improved early detection and timely treatment as well.

References

  1. "Open-Label Extension Study of Recombinant Human Arylsulfatase A (HGT-1111) in Late Infantile MLD". ClinicalTrials.gov. 21 May 2008. Retrieved 3 November 2021.
  2. "Multicenter Study of HGT-1110 Administered Intrathecally in Children With Metachromatic Leukodystrophy (MLD) (IDEAMLD)". ClinicalTrials.gov. 13 January 2012. Retrieved 3 November 2021.
  3. "Sick child's parents dump $1.75m outside drugs firm". The Sydney Morning Herald. 16 July 2009. Retrieved 3 November 2021.
  4. Jonckheere, An I.; Kingma, Sandra D. K.; Eyskens, François; Bordon, Victoria; Jansen, Anna C. (2023-09-01). "Metachromatic leukodystrophy: To screen or not to screen?". European Journal of Paediatric Neurology. 46: 1–7. doi:10.1016/j.ejpn.2023.06.005. ISSN 1090-3798.


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