How KYGEVVI was studied
78 treated patients with TK2d symptom onset ≤12 years of age were compared to a control group of 78 untreated patients
Clinical trials usually include two groups—one given the drug being studied and one given a placebo.
KYGEVVI data were collected differently. Researchers conducted a clinical trial where patients received KYGEVVI. Researchers also reviewed past health records of patients who had taken pyrimidine nucleosides. Pyrimidine nucleosides are building blocks needed for mitochondrial DNA in the muscles. An expanded access program allowed other patients to receive KYGEVVI. Results from patients who received KYGEVVI were compared to results from patients who did not receive any treatment.
To study KYGEVVI, researchers compared 2 groups:
Treated patients (n=78)
Health record review
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Open-label clinical trial
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Expanded access program
Patients were treated for 1 day to 12 years, with a median duration of 4 years
Control group of untreated patients (n=78)
Health record review
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Medical literature review
Studies found that KYGEVVI may help people with early-onset TK2d live longer
To see if KYGEVVI can help patients with early-onset TK2d live longer, researchers matched and compared patients who received treatment to untreated patients to determine improvement in survival time after starting treatment.
96% (n=75/78) of treated patients with TK2d were alive at the end of the study
64% (n=50/78) of untreated patients were alive at the end of the study
KYGEVVI safety
Take a look at the safety profile for KYGEVVI.