Mucopolysaccharidosis Iva

Top drugs studied for this disease

All Approvals Safety alerts Label changes Trial results Research Guidance Withdrawals

EMA approval · 2025-10-23
EMA Authorised: Vimizim (recombinant human n-acetylgalactosamine-6-sulfatase) — BioMarin International Limited
The European Medicines Agency authorised Vimizim for the treatment of Mucopolysaccharidosis IVA (Morquio A) in 2021; Vimizim is an enzyme re…