Aneal Khan’s Post

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Discovery DNA / MAGIC Clinic in Calgary

Putting reporting of rare diseases into perspective. So in this report a drug is toted as prolonging the life in a patient with a neurological disorder. The drug mentioned is a form of cholic acid which is a bile acid we make in our livers every day. It has never been proven to have any effect on neurological disease (how it would get in the brain is beyond explanation) and it has not impacted neurological outcomes in people with a CNS disorders like Zellweger spectrum (which is what I suspect the person has). No claim is made that it prolongs life by the manufacturer - that’s a fake claim in the report. But the reporter gives the impression that it’s life saving therapy for $600,000 a year that everyone is denying. So the reporters claim is false. I’m all for value and when a drug is expensive sometimes it’s needed - when it does something significant. It is doubtful it will do anything here except modify some liver results. Why the reporter didn’t talk to someone who knows about the drug - I don’t know. It brings to discussion how we pay for rare disease drugs - do we pay to correct just lab values or when there is a demonstrable impact on health ? https://lnkd.in/grkGNprn

Kitchener family says their 10-year-old needs life-saving drug that cost $600,000

Kitchener family says their 10-year-old needs life-saving drug that cost $600,000

kitchener.ctvnews.ca

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