INBORN ERRORS OF METABOLISM DISORDERS
Clinical trials for INBORN ERRORS OF METABOLISM DISORDERS explained in plain language.
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Half-Matched stem cells give bone marrow a jump start for High-Risk patients
Disease control AVAILABLEThis program offers extra stem cells from a half-matched family donor to patients who are getting a cord blood transplant for serious blood cancers or immune disorders. The goal is to help the bone marrow recover more quickly while the cord blood cells take over permanently. It i…
Matched conditions: INBORN ERRORS OF METABOLISM DISORDERS
Sponsor: Joanne Kurtzberg, MD • Aim: Disease control
Last updated Jun 27, 2026 07:58 UTC
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A cartoon-style program may help children with rare diseases understand their condition and boost mental health
Symptom relief Recruiting nowThis trial tests a short, child-friendly psychoeducation program called Education & Care in RARE for children and teens aged 5–20 with any rare disease. The program uses simple, resource-oriented materials to teach kids about their condition and build coping skills. Researchers w…
Matched conditions: INBORN ERRORS OF METABOLISM DISORDERS
Phase: NA • Sponsor: Medical University of Vienna • Aim: Symptom relief
Last updated Jul 30, 2026 00:00 UTC