https://nvp-tnks656inhibitor.c....om/distinct-manageme
Metachromatic leukodystrophy (MLD) is an autosomal recessive disorder caused by lacking arylsulfatase A (ASA) activity and described as neurological participation that results in extreme disability and early death. We examined the safety and tolerability of intrathecally delivered recombinant individual ASA (rhASA; SHP611, now TAK-611) in children with MLD (NCT01510028). Additional endpoints included improvement in cerebrospinal fluid (CSF) sulfatide and lysosulfatide l
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