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This issue covers August 3, 2026 to August 9, 2026 and was published August 10, 2026. Read the latest week

The week in rare disease

to · Issue 16

Orzeyful approved, and 194 trial changes across 59 conditions

In the week of Aug 3rd we recorded 194 changes to rare disease trials on ClinicalTrials.gov, 1 FDA decision, and 3 trials that opened to new patients.

By Jason Hunter · Published Monday, , covering the week that ended the day before

  1. AUG3Mon24trial changes
  2. AUG4Tue14trial changes
  3. AUG5WedApprovalOrzeyful, plus 8 changes
  4. AUG6Thu54trial changes
  5. AUG7Fri18trial changes
  6. AUG8Sat33trial changes
  7. AUG9Sun43trial changes
FDA and moneyTrial changesNew guidesGreen tiles mark an approval or a trial opening, grey a refusal or a trial closing, an outline something still to come

Trials that opened and closed

Status changes recorded on ClinicalTrials.gov this week, 37 in all

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Past weeks

Every issue stays here at its own address. The archive begins with the first week we recorded, the week of April 20th, 2026.

  1. #28Week of October 26thVoceryq is approved for RRP, three myasthenia gravis trials close to new patients, and Kite's CAR-T reaches past myeloma1 3
  2. #27Week of October 19thUltomiris approved for IgA nephropathy, 7 new guides1 7
  3. #26Week of October 12thEnspryng approved for Thyroid eye disease, 10 new guides1 10
  4. #25Week of October 5th5 new guides5
  5. #24Week of September 28thCamzyos approved for Obstructive hypertrophic cardiomyopathy, adolescents, Emcitate approved for MCT8 deficiency, 9 new guides2 9
  6. #23Week of September 21stAtebrioz approved for Fibrodysplasia ossificans progressiva, Gazyva approved for Idiopathic nephrotic syndrome, 9 new guides3 9
  7. #22Week of September 14thAqneursa approved for Ataxia-telangiectasia, Fayuvi approved for Sanfilippo syndrome type A, 9 new guides2 9
  8. #21Week of September 7thIsembyld approved for Spinal muscular atrophy, Joenja approved for Activated PI3K delta syndrome (APDS), children 4 to 11, 5 new guides2 5
  9. #20Week of August 31stZanvastro approved for Alexander disease1
  10. #19Week of August 24thBesremi approved for Essential thrombocythemia, Mimrylo approved for Polycythemia vera, 1 new guide4 1
  11. #18Week of August 17thGenglycos approved for Glycogen storage disease type 1a, Pasatru approved for Fibrodysplasia ossificans progressiva2
  12. #17Week of August 10thTrial changes across the conditions we follow
  13. #16Week of August 3rdOrzeyful approved for Narcolepsy type 11
  14. #15Week of July 27thTrial changes across the conditions we follow
  15. #14Week of July 20thTrial changes across the conditions we follow
  16. #13Week of July 13thFabhalta approved for IgA nephropathy1
  17. #12Week of July 6thTrutakna approved for IgA nephropathy1
  18. #11Week of June 29thTrial changes across the conditions we follow
  19. #10Week of June 22ndTrial changes across the conditions we follow
  20. #9Week of June 15thTrial changes across the conditions we follow
  21. #8Week of June 8thTrial changes across the conditions we follow
  22. #7Week of June 1stTrial changes across the conditions we follow
  23. #6Week of May 25th1 new guide1
  24. #5Week of May 18th1 new guide1
  25. #4Week of May 11th1 new guide1
  26. #3Week of May 4th4 new guides4
  27. #2Week of April 27th4 new guides4
  28. #1Week of April 20th2 new guides2