Globoid cell leukodystrophy | Article about globoid cell leukodystrophy by The Free Dictionary
leukodystrophy (redirected from globoid cell leukodystrophy)
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A condition thought to result from an inborn error of metabolism and characterized by progressive degeneration of the white matter of the cerebrum, or by defective buildup of myelin.
References in periodicals archive
Globoid cell leukodystrophy
(GLD) or Krabbe disease (KD) is an inherited neurodegenerative lysosomal enzyme disorder affecting the central and peripheral nervous systems.
The Company plans to enroll 12 patients in the ongoing Phase 2 study in inherited metabolic disorders, which include cALD, metachromatic leukodystrophy and globoid cell leukodystrophy
. The study previously enrolled patients with Hurler syndrome.
Orchid will also acquire the rights to exclusively license three additional preclinical programmes from Telethon/Ospedale San Rassaele once clinical proof of concept studies for mucopolysaccharidosis type 1 (MPS1 or Hurler syndrome), chronic granulomatous disease (CGD), and globoid cell leukodystrophy
(GLD) are complete.