Alkaptonuria

• CLINICAL_TRIALS research · 2026-03-31
  Clinical Trial Update: Study of Alkaptonuria

  An NHGRI-sponsored clinical trial studying alkaptonuria is currently recruiting participants.

• CLINICAL_TRIALS trial_result · 2025-11-28
  Clinical Trial Update: Evaluation of TYR Sphere in France [NA]

  A completed clinical trial in France evaluated TYR Sphere for tyrosinemia and alkaptonuria.

• CLINICAL_TRIALS trial_result · 2025-03-26
  Clinical Trial Update: Questionnaire Follow=up Study Sonia 2

  A completed follow-up questionnaire study (Sonia 2) related to Alkaptonuria, conducted by Liverpool University Hospitals NHS Foundation Trus…

• CLINICAL_TRIALS trial_result · 2021-04-22
  Clinical Trial Update: Nitisinone (NTBC) In Different Age Groups Of Patients With Alkaptonuria [Phase 2, Phase 3]

  Phase 2/3 trial of nitisinone (NTBC) in different age groups for alkaptonuria has completed with posted results.

• EMA approval · 2020-09-18
  First treatment for rare metabolic disorder alkaptonuria

  EMA announces the first approved treatment for alkaptonuria, a rare metabolic disorder, marking a pivotal milestone for patients affected by…

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• CLINICAL_TRIALS other · 2018-05-31
  Clinical Trial Update: Suitability of Nitisinone in Alkaptonuria 2 [Phase 3]

  A Phase 3 trial update notes the suitability of nitisinone for alkaptonuria. The study is sponsored by the University of Liverpool and the c…

• CLINICAL_TRIALS trial_result · 2014-06-11
  Clinical Trial Update: Dose Response Study of Nitisinone in Alkaptonuria [Phase 2]

  A Phase 2 dose-response study evaluating nitisinone in alkaptonuria has completed.

• CLINICAL_TRIALS trial_result · 2011-01-19
  Clinical Trial Update: Long-Term Study of Nitisinone to Treat Alkaptonuria [Phase 2]

  A Phase 2 long-term trial of nitisinone for alkaptonuria has completed, with results posted in 2011.