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enzyme replacement therapy.
It also dramatically lowers the cost of enzyme replacement therapy.
Enzyme replacement therapy and gene therapy are new treatments in development for patients with MLD [11].
Approved therapies were enzyme replacement (N = 10), substrate reduction (N = 1), and small molecules (N = 3).
This year, Alexion anticipates U.S. approval for Strensiq, an enzyme replacement therapy for HPP.
Patients who have lysosome diseases like mucolipidosis can be treated with enzyme replacement therapy.
Approximately 80% of the patients have received enzyme replacement therapy, hematopoietic stem cell transplantation, or both.
Oral pancreatic enzyme replacement therapy (PERT) with pancreatin produces unsatisfactory results.
Enzyme replacement and substrate deprivation therapies are currently used to treat Gaucher disease.
Seventy-eight percent (582/742) of registry patients have received enzyme replacement therapy.
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The acquisition also gives Genzyme new methods for making proteins for enzyme-replacement therapies.
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