Rare disease news

FDA approvals, research breakthroughs, clinical trials, and advocacy updates

Curated and summarized by AI for patients and caregivers

🔍
AllDrug approvalsClinical trialsResearchGrants & fundingAdvocacy & policyPipeline
Show:All newsBreaking onlyImportant & breaking
Date:7 days30 days90 daysAll time

2 articles from the last 90 days matching "blood disorders"

Clinical trialUNITERAREYesterday

New Recruiting Trial: Study of Ianalumab in Adults With Primary Immune Thrombocytopenia (ITP) and Warm-antibody Autoimmune Hemolytic Anemia (wAIHA) Who Have Previously Benefited From Ianalumab

Novartis is looking for adults who have immune thrombocytopenia (ITP) or warm-antibody autoimmune hemolytic anemia (wAIHA) — two blood disorders where the immune system attacks blood cells — and who previously did well on a drug called ianalumab. This new study will test whether ianalumab continues to work for these patients. The trial is expected to start in April 2026.

WHY IT MATTERSThis trial is recruiting adults who previously responded to ianalumab for ITP or wAIHA, offering a potential path to continued access for patients whose symptoms improved on this treatment.
💬 Ask your doctorPrimary Immune Thrombocytopenia (ITP)Warm-antibody Autoimmune Hemolytic Anemia (wAIHA)Read →
Clinical trialUNITERAREApr 5

New Recruiting Trial: Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms

Researchers are testing a new drug called pacritinib in people ages 12 and older who have myelodysplastic syndromes or myelodysplastic/myeloproliferative neoplasms — rare blood disorders where the bone marrow doesn't make enough healthy blood cells. The drug works by blocking several proteins that may be causing these diseases. This is an early-stage study sponsored by the National Cancer Institute that will start recruiting patients in April 2026.

WHY IT MATTERSThis trial offers patients with myelodysplastic syndromes access to a novel multi-targeted kinase inhibitor that may address disease mechanisms not covered by current standard treatments, with enrollment beginning in April 2026.
👁 Watch this spaceMyelodysplastic SyndromesMyelodysplastic/Myeloproliferative NeoplasmsRead →

Get personalized rare disease news

Follow your conditions to see news about the diseases that matter to you — FDA approvals, trial openings, and research breakthroughs.

Create free account →Browse diseases