BBC article about successful immune system med for rare genetic ADPS disorder

Leniolisib has a list price of £352,000 a year, but was approved as cost effective by the health regulator NICE after the NHS negotiated a substantial, confidential discount.
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For reference: Daratumumab is a lot cheaper, and will probably become even cheaper when the patents runs out.

It is interesting to see how testing for APSD is quite strongly recommended, even before this treatment was available.

For Huntington’s disease (HD), which is inherited the same way, progressive, and eventually fatal, there is much more focus on everyone doing what’s right for them. I’m assuming the scales shift when you’ve got at least some treatment options like for APSD, and knowing if you’re positive might actually make a difference by getting access to treatments. The main concern for HD is if you want to test before you get children, and what a positive test might mean for children you’ve already got.
 
Prof Sergey Nejentsev from the University of Cambridge who led the research that discovered APDS said: "As soon as we understood the cause of APDS, we immediately realised that certain drugs could be used to inhibit the enzyme that is activated in these patients.

Leniolisib does precisely that. I am delighted that we finally have a treatment which will change the lives of APDS patients."

Leniolisib has a list price of £352,000 a year, but was approved as cost effective by the health regulator NICE after the NHS negotiated a substantial, confidential discount.

NICE estimates the drug could benefit up to 50 patients over the age of 12 in England.
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