Retrophin recruiting for PKAN drug study

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May 4, 2017

The NBIA Disorders Association posts the following announcement for informational purposes only. While the organization supports and encourages the discovery of treatments for NBIA individuals and willingly posts information concerning research studies (such as questionnaires and clinical trial enrollment), we do not endorse specific studies. Nor do we advise NBIA individuals or their families to take part in a particular study. Rather, we believe that those decisions are best made by affected individuals and/or their families, in collaboration with their doctors.

Retrophin Inc. has begun to recruit patients for its planned clinical trial for PKAN patients.

The company plans to test a drug, fosmetpantotenate, the new name for RE-O24, to see if it can help patients with the most common NBIA disorder, PKAN. Retrophin had hoped to begin the study late last year, but a manufacturing problem caused a delay until now.

In the clinical trial, which takes its name from the drug and is being called the FORT Study, Retrophin plans to recruit 82 people diagnosed with PKAN in North America and Europe. Eligible patients must range in age from 6 to 65, be able to breathe without a ventilator, must be off of deferiprone for 30 days and cannot have had a deep brain stimulation device implanted within the past six months.

For information about the study, trial sites and screening requirements for participants, visit Information about the study is also available on


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