罕见病药物氨己烯酸片效果好不好

(Vigabatrin) is a synthetic derivative of gamma-aminobutyric acid (GABA). This product increases the concentration of GABA in the brain by irreversibly inhibiting GABA aminotransferase.

Vigabatrin is used as adjuvant therapy in patients who are refractory to other antiepileptic drugs, especially those with partial seizures (mainly used to control complex partial seizures). This product may aggravate absence seizures or myoclonic seizures. Vigabatrin tablets may also be used in infants with West syndrome (infantile spasms), which is generally unresponsive to conventional antiepileptic drugs. As an auxiliary treatment drug, Vigabatrin tablets can achieve better efficacy. Effective in the treatment of newly diagnosed patients when used alone

Are the rare disease drug vigabatrin tablets effective?

A clinical trial explores the therapeutic effect of a rare disease drug (Vigabatrin) on refractory epilepsy in children. The drug-increasing test method was used to observe the efficacy and safety of the drug on 25 children. Two months after taking the medicine, the total effective rate was 68.0%. There were 24 patients who took the medicine for ≥4 months, and the total effective rate was 70.8% (21/24). After administration of vigabatrin, phenobarbital blood concentrations are lower than before administration. The serum alanine aminotransferase level was lower than before taking the medicine. No side effects were seen in 32 cases. 2 cases developed mild dizziness and drowsiness in the early stage after taking the medicine, which were relieved by themselves; 1 case developed severe restlessness and terminated treatment. Observation results show that Vigabatrin tablets are more effective in treating refractory epilepsy in children and have less adverse side effects.

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