ALDURAZYME (laronidase), enzyme replacement

RARE DISEASE - Update

Opinions on drugs - Posted on May 23 2017

Re-assessment of the actual benefit and of the improvement in actual benefit

ALDURAZYME has marketing authorisation for long-term enzyme replacement therapy in patients with mucopolysaccharidosis type I (MPS I), to treat the non-neurological manifestations of the disease.
It is the only enzyme replacement therapy and the only treatment option for patients who are not eligible for haematopoietic stem cell transplant.
The results of clinical trials imply stabilisation of respiratory function, endurance, sleep quality and quality of life in patients with an intermediate or attenuated form of MPS I.
ALDURAZYME has no effect on the neurological signs of MPS I and little efficacy on osteoarticular signs, ophthalmic signs and valve disease.

Substantial

important

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