"As a community, we are delighted that Lumos Pharma is taking the lead in developing a potential treatment for our CTD boys."
Linda Cooper - Parent
Director of the Association for Creatine Deficiencies

"Currently, there is no effective treatment for our patients with creatine transporter deficiency. It is our sincere hope that Lumos’ treatment will improve the outcome of these patients and, if started early, possibly completely prevent intellectual disability."

Nicola Longo, MD, Ph.D.
Chief, Division of Medical Genetics University of Utah

“I look forward to working with Lumos and the NIH Therapeutics for Rare and Neglected Diseases program in advancing therapeutics for CTD patients."

Gajja Salomons, Ph.D.
Professor of Molecular Biology of Neurometabolic disorders VU Medical Center, Netherlands

Get Involved!

Get plugged in! Find out more about how you can make an impact and help those affected by CTD.

What is CTD?

Creatine Transporter Deficiency is an x-linked inborn error of creatine metabolism that results in an absence of creatine in the brain. This causes severe global developmental delay, mental retardation, Autistic behavior, and expressive speech deficits.

How Can I help?

Lumos Pharma, with the help of the CTD community, hopes to provide a much needed therapeutic for those afflicted with CTD.

Learn about LUM-001

LUM-001 has shown great therapeutic potential for the treatment of CTD in a validated animal model. Lumos Pharma, in collaboration with the National Institutes of Health are conducting the necessary studies in order to initiate clinical trials in human patients.