Currently, there is no disease modifying therapy. Blood pressure and renal function monitoring is essential considering the high prevalence of kidney failure in patients. Renal replacement therapy including transplant has shown to improve overall clinical outcome in patient with milder neurological involvement. Patients may also develop mild to intractable epilepsy which may require careful EEG monitoring and medication titration. Spasticity and dystonia have been reported in some individual with infantile onset disease; in these patients, targeted botulin toxin injection could be considered.
RMND1