Disease index

mucopolysaccharidosis IVA

Metabolic · 2014–2014

1decision
1medicine
1carried consumer input
91individual submissions

Every decision

MedicineMeetingOutcomeConsumer input
elosulfase alfa Treatment of mucopolysaccharidosis IVA (Morquio A Syndrome), an inherited lysosomal storage disorder characterised by absence of the enzyme… Nov 2014 Not recommended 91 individuals

Conditions are classified by a language model from the indication text of each submission, so an individual label here can be wrong. Consumer input is read from the document's own consumer comments section: 1 of 1 decisions since 2014 carried some. Silence means no comment reached the committee through that facility, not that nobody was affected. More on how this was built.