Orphan Drug Designations

The following drugs have received orphan drug designation from the FDA for atypical hemolytic-uremic syndrome. Orphan designation reflects regulatory interest and does not indicate approval for treatment.

Gene therapy approaches for atypical hemolytic-uremic syndrome have been reported in the published literature.

247 publications have been identified in PubMed for atypical hemolytic-uremic syndrome. Research spans Case Report / Case Series (38%), Review / Meta-Analysis (25%), and Clinical Trial Publication (12%).

Recent Publications

• Java A (2026). [PMID: 41649885](https://pubmed.ncbi.nlm.nih.gov/41649885/). *Clin J Am Soc Nephrol*. [Gene Therapy / Novel Therapeutics]

• Chen X (2026). [PMID: 41866478](https://pubmed.ncbi.nlm.nih.gov/41866478/). *BMC Nephrol*. [Case Report / Case Series]

• Orozco-Leal G (2026). [PMID: 40844834](https://pubmed.ncbi.nlm.nih.gov/40844834/). *Nephrol Dial Transplant*. [Epidemiology / Natural History]

• Valdez-Thomas M (2026). [PMID: 41236470](https://pubmed.ncbi.nlm.nih.gov/41236470/). *JACC Case Rep*. [Case Report / Case Series]

• Tsiamis N (2026). [PMID: 41874770](https://pubmed.ncbi.nlm.nih.gov/41874770/). *CEN Case Rep*. [Case Report / Case Series]

• Risly NMM (2026). [PMID: 42169153](https://pubmed.ncbi.nlm.nih.gov/42169153/). *J Med Case Rep*. [Case Report / Case Series]

• Collins C (2026). [PMID: 41953852](https://pubmed.ncbi.nlm.nih.gov/41953852/). *SAGE Open Med Case Rep*. [Case Report / Case Series]

• Yanagidani H (2026). [PMID: 41680667](https://pubmed.ncbi.nlm.nih.gov/41680667/). *BMC Nephrol*. [Case Report / Case Series]

• Alkhaldi A (2026). [PMID: 41987101](https://pubmed.ncbi.nlm.nih.gov/41987101/). *BMC Nephrol*. [Case Report / Case Series]

• Krajina L (2026). [PMID: 42144300](https://pubmed.ncbi.nlm.nih.gov/42144300/). *Transplant Proc*. [Case Report / Case Series]