Is there a cure for Morquio's Syndrome?

Answer 1

Morquio Syndrome is an inherited metabolic disease. Symptoms include an enlarged heart, dwarfism, abnormal skeletal development and knock-knees. There is no cure for Morquio Syndrome but it can be treated with a drug called Vimizim.

Answer 2

There is a treatment but no cure.Biomarin is developing an Enzyme Replacement Therapy for Morquio. They are expected to announce Phase 1/2 results shortly and from initial data (1mg/kg) efficacy and safety seem promising. If everything continues to look good a Phase 3 study could begin late '10/early '11 and if those results are positive the therapy could be on the market possibly as early as late '12. Getting into a Phase 3 trial would probably be the quickest way to get treatment though one could be randomized to Placebo. The possibility of compassionate use/early access exists as well.There was another company in earlier stages of development for an Enzyme Replacement but I believe they had (financial) problems.