Barth syndrome (FDA label)
Who: Adults and children with Barth syndrome who weigh at least 30 kg, on Forzinity.
Timing: Same time each day. Inject into the belly, at least 2 inches from the navel, or the outer thigh, rotating sites daily.
Note: 40 mg is 0.5 mL of the 80 mg/mL solution. Subcutaneous only, never IV. Skip a missed dose rather than doubling. Community doses sit far below this. No trial tested 1 to 10 mg a day except one 4 mg heart-failure arm.