My sister's CK levels have been very high (387 in 2015, 311 in 2015, etc.) and her progression has been very slow relative to the literature that I have read. However, her motor function has steadily declined. It started in her lower extremities and she is no longer able to walk or stand. Over the past year, she lost significant strength in her arms, to the point that she could not lift a cup to drink, brush her hair, etc. I posted on another thread that she started having severe systemic itching about 1 year ago (awful, to the point she could not sleep). We tried all types of creams and OTC therapies, but nothing completely resolved the itching. She had blood work done a few months ago to look at renal and liver function and all was fine except high normal ALT (liver function test). The ordering physician started her on cholesteramine and within 2 weeks, she started to regain strength in her arms and now (2 months later) she can lift a cup and drink without spilling, brush her hair, etc. She still has the itching, but not as severe. I have been hesitant to post more on this because it was coincidental that she received the cholesteramine and I have never heard of any regression of symptoms with this disease. I have read everything I can and other than some research done on bile acid metabolism, I can't find any connection. Even AMX0035 reported delay in progress in 38% of patients (I realize it is not the same as cholesteramine, but it is a combo drug of sodium phenylbutyrate and taurursodiol). I am waiting to take her back to her neurologist (appointment in January).