FANCONI ANEMIA
Clinical trials for FANCONI ANEMIA explained in plain language.
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Gene therapy for rare blood disease tracked for 15 years
Disease control OngoingThis study follows 9 people with Fanconi Anemia who received a one-time gene therapy to fix a faulty gene. Researchers will monitor their health and blood counts for 15 years to see if the treatment remains safe and effective. No new treatment is given during this follow-up perio…
Matched conditions: FANCONI ANEMIA
Sponsor: Rocket Pharmaceuticals Inc. • Aim: Disease control
Last updated May 22, 2026 13:55 UTC
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Gene therapy for rare blood disease shows promise in Long-Term Follow-Up
Disease control ENROLLING_BY_INVITATIONThis study checks the long-term safety and effectiveness of a gene therapy called RP-L102 for people with Fanconi Anemia, a rare genetic condition that causes bone marrow failure and raises cancer risk. About 14 patients who already received RP-L102 in earlier studies will be fol…
Matched conditions: FANCONI ANEMIA
Sponsor: Rocket Pharmaceuticals Inc. • Aim: Disease control
Last updated Apr 29, 2026 15:04 UTC
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Glow-in-the-Dark mouth scans could catch cancer early
Diagnosis OngoingThis study tests whether special lights and cameras can help find early signs of mouth cancer in people at high risk. Participants include those with mouth spots (leukoplakia, erythroplakia), a history of head and neck cancer, or conditions like Fanconi anemia or heavy tobacco us…
Matched conditions: FANCONI ANEMIA
Phase: NA • Sponsor: M.D. Anderson Cancer Center • Aim: Diagnosis
Last updated May 22, 2026 14:04 UTC