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Metazym is an experimental recombinant enzyme that was studied in patients with late infantile metachromatic leukodystrophy,[1] but found to be ineffective under the conditions of that trial. A subsequent clinical trial is ongoing.[2] The drug became a source of controversy when a family attempted to purchase the drug for their child before it was approved.[3] 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.[4]
References edit
- ^ "Open-Label Extension Study of Recombinant Human Arylsulfatase A (HGT-1111) in Late Infantile MLD". ClinicalTrials.gov. 21 May 2008. Retrieved 3 November 2021.
- ^ "Multicenter Study of HGT-1110 Administered Intrathecally in Children With Metachromatic Leukodystrophy (MLD) (IDEAMLD)". ClinicalTrials.gov. 13 January 2012. Retrieved 3 November 2021.
- ^ "Sick child's parents dump $1.75m outside drugs firm". The Sydney Morning Herald. 16 July 2009. Retrieved 3 November 2021.
- ^ 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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