FIELD: biotechnology.
SUBSTANCE: invention relates to biotechnology and is a composition for treating metachromatic leukodystrophy (MLD) containing a purified protein, which is recombinant human aryl sulphate A (rhASA), with an amino acid sequence at least 70% identical to SEQ ID NO: 1, where the purified rhASA protein is characterized by one or more types of proteoglycans selected from i) from 15% to 25% of neutral recombinant ASA protein (neutral ASA protein), ii) from 35% to 45% of sialylated recombinant protein ASA (ASA protein with sialic acid), iii) from 23% to 33% of mannose-6-phosphated recombinant protein ASA (ASA protein with M6P), iv) from 1% to 10% N-acetyl-glucosamine-mannose-6-phosphated recombinant protein ASA (ASA protein capped by M6P) and v) from 5% to 15% of hybrid recombinant protein ASA (hybrid protein ASA).
EFFECT: invention provides higher clinical effectiveness in metachromatic leukodystrophy.
26 cl, 22 dwg, 11 tbl, 7 ex
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