Orchard will also acquire rights to exclusively license three additional preclinical programmes from Telethon/Ospedale San Raffaele upon completion of clinical proof of concept studies for mucopolysaccharidosis type 1 (MPS1 or Hurler syndrome), chronic granulomatous disease (CGD) and globoid cell
Krabbe disease (globoid cell
leukodystrophy) is an often-fatal lysosomal storage disease caused by the deficiency of the enzyme galactocerebrosidase (GALC; (8) EC 126.96.36.199), resulting in the loss of the myelin sheath in the central nervous system (1, 2).
There are various conditions, which affect spinal cord such as degenerative myelopathy, spinal dysraphism, spina bifida, myelodysplasia, metabolic globoid cell
leukodystrophy, neoplasia, immune mediated conditions, tetanus, vascular anomalies and traumatic conditions (Birchard and Sherding, 1994).
(9) Amongst those involving predominantly white matter can be further categorised as predominant involvement of periventricular white matter as in Metachromatic leukodystrophy, Classic X-linked adrenoleukodystrophy, Globoid cell
leukodystrophy (Krabbe disease), vanishing white matter disease and those with predominant subcortical white matter involvement as Van Der Knaap disease.