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Description
Increasing the interval between successive injections and even eliminating the need for additional intervention is an important goal of therapy
The MTHFR gene polymorphism is linked to onset, clinical symptoms, prevalence as well as response to treatments
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The novel triple-agonist mechanism means less long-term safety data is available
Menkes disease typically presents in boys aged 23 months, with the neurodevelopmental delay and degeneration, seizures and failure to thrive
