INHERITED METABOLIC DISORDERS (IMD)
Clinical trials for INHERITED METABOLIC DISORDERS (IMD) explained in plain language.
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Gentler transplant offers hope for kids with rare, Life-Limiting diseases
Disease control Recruiting nowThis study is testing a less intense chemotherapy regimen before a stem cell transplant for children and young adults with inherited disorders like sickle cell disease, immune deficiencies, and metabolic diseases. The goal is to see if this gentler approach helps donor stem cells…
Matched conditions: INHERITED METABOLIC DISORDERS (IMD)
Phase: PHASE2 • Sponsor: Paul Szabolcs • Aim: Disease control
Last updated Apr 03, 2026 14:43 UTC
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Gentler transplant offers hope for rare disease patients
Disease control Recruiting nowThis study is collecting information on a less intense stem cell transplant method for people with inherited disorders like immune deficiencies, sickle cell disease, or metabolic diseases. It aims to see how well patients recover, how often the transplant is successful, and how l…
Matched conditions: INHERITED METABOLIC DISORDERS (IMD)
Sponsor: Paul Szabolcs • Aim: Disease control
Last updated Mar 31, 2026 12:11 UTC