This is a consensus article that sews together bunches of facts. It seems to answer many of the questions I am hearing from newly diagnosed ALD/AMN patients and their caregivers. Have a look. Keep it straight. Brad https://journals.lww.com/neurotodayonline/fulltext/2022/10200/new_consensus_document_provides_practical_guidance.4.aspx
Tag Archives: orphan disease
Right to Try?
‘Remember her as the badass she was.’ The first bit of the title caught my attention, as I expect that it was intended to do. I had a look. I like to stir the pot from time to time, this one delivered. Expanded Access always seemed like a good idea to me, I mean ifContinue reading “Right to Try?”
Advocacy for Rare Disease Accelerated Approval
Today, the The Rare Disease Company Coalition sent this letter to committee chairs in both houses of the U.S. Congress, reminding them of the importance of accelerated approval of therapeutics to counter rare diseases. I applaud the Coalition for taking this important action. I encourage everyone to contact your member of congress to backstop thisContinue reading “Advocacy for Rare Disease Accelerated Approval”
Walking the Rails 