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What is an Aldose reductase inhibitor?
Aldose reductase inhibitors make the aldose reductase protein work slowly and have been developed for the treatment of diabetes. Aldose reductase is a protein that uses glucose (a type of sugar) to make sorbitol. When glucose levels are high (e.g. diabetes), the aldose reductase protein starts to work to remove the glucose from tissues that are not sensitive to the action of insulin, particularly the optic lens, the peripheral nerves and the glomerulus. This leads to retinopathy and neuropathy in diabetic patients.
How can this help people living with PMM2-CDG?
Patients with PMM2-CDG have imbalances in sugar metabolism pathways, including the aldose reductase ability to work. The company Applied Therapeutics has been developing an aldose reductase inhibitor, called AT-007. Initial data in fibroblast cell lines derived from patients with PMM2-CDG demonstrates that AT-007 increases PMM2 activity and may provide therapeutic benefit in PMM2-CDG patients. Applied Therapeutics plans to initiate a clinical study in PMM2-CDG patients in 2021.
Publications in CDG are constantly growing. We have selected articles of special interest for you, see below. Not all are open access, thus if you wish to access the full information, please contact us at https://worldcdg.org/contact
