Update: the FANCA Gene Therapy Trial

The global Fanconi anemia gene therapy program is now open in the USA and Europe. In Europe, a new study, known as FANCOLEN-2, is available at CIEMAT/Hospital del Niño Jesús in Madrid, Spain. In the USA, a similar study is now available at Stanford University. The patients already recruited for this study will begin undergoing therapy in the spring of 2019.

If you are interested in participating in the trial at Stanford this fall, the first step is to reach out to get more information. At FARF, we will connect you to those running the study so you can learn more.

Both the US and European programs are sponsored by Rocket Pharma (New York, USA). Assistance will be available for families to help with travel, housing accommodations, and other support., both for initial treatment and subsequent follow-up visits.

Categories: Announcements

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