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

3 articles matching "mds"

Clinical trialUNITERAREApr 3

New Recruiting Trial: A Single-arm, Prospective Study of a Cladribine-Bridged LABU Conditioning Regimen in Allogeneic Hematopoietic Stem Cell Transplantation for Relapsed/Refractory MDS/AML in Elderly Patients

Researchers are testing a new treatment approach for elderly patients with a type of blood cancer (MDS or AML) that has come back or stopped responding to previous treatments. The study uses a combination of chemotherapy drugs followed by a stem cell transplant from a donor to try to help patients recover. This trial is now actively looking for patients to participate.

WHY IT MATTERSThis trial offers elderly patients with relapsed or refractory MDS/AML a potentially less toxic conditioning regimen before stem cell transplant — cladribine-bridged LABU may improve tolerability compared to standard high-dose approaches in this vulnerable population.
You can act on thisMyelodysplastic Syndrome (MDS)Acute Myeloid Leukemia (AML)Read →
Clinical trialUNITERAREApr 3

New Recruiting Trial: A Study to Compare Elritercept With Epoetin Alfa to Treat Anemia in Adults With Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) Who Need Regular Blood Transfusions

Researchers are testing a new drug called elritercept to see if it works better than a standard treatment (epoetin alfa) for anemia in adults with myelodysplastic syndromes (MDS)—a blood disorder where the bone marrow doesn't make enough healthy blood cells. People in this study have low, very low, or intermediate risk MDS and need regular blood transfusions. The trial is now accepting patients.

WHY IT MATTERSThis Phase 3 trial is actively recruiting adults with MDS who require transfusions, offering access to an investigational treatment that may reduce their dependence on blood transfusions.
You can act on thisMyelodysplastic SyndromesRead →
PolicyPUBMEDMar 26

Global Access to IMD Drugs: Bridging the Gap Between High-Income Countries and Latin America.

People with inherited metabolic diseases (IMDs) in Latin America struggle to get life-saving medicines that are more available in wealthy countries. This article looks at why these medicines are hard to find in places like Chile and suggests ways to make them more accessible to everyone who needs them, no matter where they live.

WHY IT MATTERSIf you or a family member has an inherited metabolic disease in Latin America, this research directly addresses the barriers preventing you from accessing treatments that patients in high-income countries can obtain.
💬 Ask your doctorInborn Errors of Metabolism (IMDs)Inherited Metabolic DiseasesRare DiseasesRead →

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