Interesting clinical trial in the US! - Leukodystrophy Su...

Leukodystrophy Support

195 posts

Interesting clinical trial in the US!

1 Reply

Dr. Scanlan from the Oregon Health & Science University is carrying out a study of sobetirome in X-ALD. He believes: “sobetirome may be beneficial to patients with ALD because it will stimulate the production of a gene product called ABCD2 which is very closely related to ABCD1, the defective gene in ALD patients, and ABCD2 can compensate for defective ABCD1. One measure of this compensation is reduction of the elevated VLCFA levels in ALD patients […] we also believe that sobetirome may arrest the progression of neurological symptoms in both cerebral ALD and AMN”. For more information about his research, please visit our research updates page: aldlife.org/news-research/r....

Your thoughts?

Read more about...

The ability to reply to this post has been turned off.

1 Reply

Also coming out of the USA this Easter weekend, New York legislature approves Aidan’s law. The law named after Aidan Seeger, who was diagnosed with ALD in 2011, requires ALD to be added to New York’s Newborn Screening Panel. Well done to all of those involved in getting the bill through!