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The safety and efficacy of Myozyme were assessed in two separate clinical trials in 39 infantile-onset patients with Pompe disease ranging in age from 1 month to 3.5 years at the time of the first infusion. [8] Myozyme treatment prolongs ventilator-free survival and overall survival. Early diagnosis and early treatment lead to much better outcomes.
A small new trial published in the journal Nature Medicine describes what would be two firsts for Parkinson's disease, if they pan out: a diagnostic test and a potential immune-based treatment ...
Avalglucosidase alfa is composed of the human GAA enzyme that is conjugated with a couple of bis-mannose-6-phosphate (bis-M6P) tetra-mannose glycans. [12] The bis-MGP of avalglucosidase alpha binds to the cation-independent mannose-6-phosphate receptor which is located on the skeletal muscles. [12]
The FDA has placed a clinical hold on Astellas Pharma Inc's (OTC: ALPMY) FORTIS Phase 1/2 trial following a serious adverse event (SAE) of peripheral sensory neuropathy in one of the trial ...
However, with this treatment, the number of substantia nigra neurons decrease so it becomes less efficient. [2] These treatments try to reduce the symptoms of the patient focusing on increasing the production of dopamine but they do not cure the disease. The new treatments for PD are in clinical trials and most of them are centered on gene therapy.
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