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

81 articles from the last 30 days matching "Research"

Clinical trialUNITERAREApr 3

New Recruiting Trial: Functional Ovarian Reserve in Sickle Cell Disease

Researchers at St. Jude Children's Research Hospital are looking for people with sickle cell disease to participate in a study about egg health and fertility. The study will examine how sickle cell disease affects the ovaries and a woman's ability to have children. This research could help doctors better understand and protect fertility in women with sickle cell disease.

WHY IT MATTERSThis trial is now recruiting patients with sickle cell disease to understand how the disease affects ovarian function and fertility — information that could lead to better fertility preservation strategies for affected women.
You can act on thissickle cell diseaseRead →
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 trialCLINICALTRIALSApr 3

Trial Now Recruiting: Understanding of Rare Inflammatory Arthritis in Comparison to Classical Inflammatory Arthritis : Tissular Observations and Immune Infiltrate Characterization : the UTOPIC Project (NCT07302074)

Researchers are looking for 100 people with rare types of joint inflammation to better understand how these conditions develop and damage joints. Some of these inflammatory arthritides occur alongside rare autoimmune diseases like systemic sclerosis, while others develop as side effects from cancer immunotherapy drugs. By studying tissue samples and immune cells from patients, scientists hope to discover why these conditions happen and potentially develop better treatments.

WHY IT MATTERSIf you have rare inflammatory arthritis, systemic sclerosis with joint involvement, or developed joint inflammation after cancer immunotherapy, this trial could help researchers understand your condition better and may lead to more targeted treatments in the future.
You can act on thisSystemic SclerosisInflammatory ArthritisImmune Checkpoint Inhibitor-Related Inflammatory ArthritisRead →
Clinical trialCLINICALTRIALSApr 3

Trial Now Recruiting: The Head and Neck Registry of the European Reference Network on Rare Adult Solid Cancers (NCT05483374)

Doctors across Europe are building a database to track rare head and neck cancers like nasopharynx cancer and salivary gland cancer. They're recruiting 13,600 patients to help them understand how these cancers develop and improve treatment. This registry will help researchers learn more about these uncommon cancers so doctors can treat patients better in the future.

WHY IT MATTERSIf you have a rare head and neck cancer like nasopharynx or salivary gland cancer, joining this registry helps European specialists understand your condition better and could improve treatment options for patients like you.
💬 Ask your doctorNasopharynx cancerNasal cavity and paranasal sinus cancerSalivary gland cancerRead →
Clinical trialUNITERAREApr 3

New Recruiting Trial: Application of Digital Twins' Technology in Patients Who Had a Stroke, With Moyamoya Disease and With Cerebral Amyloid Angiopathy (CAA) During the Secondary Prevention Phase: A Proof of Concept Using a Randomized Control Trial (Clinical Study 6, STRATIF-AI Project)

Researchers are testing a new technology called 'digital twins' to help prevent second strokes in patients with three rare brain conditions: moyamoya disease, cerebral amyloid angiopathy, and previous stroke history. A digital twin is a computer model of a patient's brain that doctors can use to predict what treatments might work best for that individual. This study will randomly assign patients to either receive care guided by this digital twin technology or standard care, and compare which approach prevents more strokes.

WHY IT MATTERSThis trial offers patients with moyamoya disease or cerebral amyloid angiopathy who have had a stroke access to personalized, AI-guided prevention strategies that could reduce their risk of future strokes.
You can act on thisMoyamoya diseaseCerebral amyloid angiopathyStrokeRead →
Clinical trialUNITERAREApr 3

New Recruiting Trial: A Phase 1 Dose-escalation Trial of KST-6051 in Participants With Advanced Solid Tumors With Kirsten Rat Sarcoma Viral Oncogene Homolog (KRAS) Mutation

Researchers are testing a new experimental drug called KST-6051 in patients with advanced solid tumors (cancers that form in organs or tissues) that have a specific genetic mutation called KRAS. This is an early-stage study (Phase 1) that will test different doses of the drug to find the safest and most effective amount. The trial is now accepting patients and is expected to start in April 2026.

WHY IT MATTERSThis trial offers patients with KRAS-mutated solid tumors access to a novel targeted therapy before it becomes widely available, potentially providing a treatment option for cancers that are often difficult to treat.
💬 Ask your doctorRead →
Clinical trialUNITERAREApr 3

New Recruiting Trial: Entrectinib as a Single Agent in Upfront Therapy for Children <3 Years of Age With NTRK1/2/3 or ROS1-FUSED CNS Tumors

Researchers at St. Jude Children's Research Hospital are testing a drug called entrectinib to treat very young children (under 3 years old) who have brain or spinal cord tumors caused by changes in specific genes called NTRK1, NTRK2, NTRK3, or ROS1. This is a Phase 2 trial, meaning they've already tested the drug in a small group and now want to see if it works well in more patients. The trial is now accepting new patients and is expected to start in April 2026.

WHY IT MATTERSThis trial offers a targeted treatment option specifically designed for infants and toddlers with NTRK- or ROS1-fused CNS tumors, a group that has historically had very limited treatment options and poor outcomes.
You can act on thisNTRK1-fused central nervous system tumorsNTRK2-fused central nervous system tumorsNTRK3-fused central nervous system tumorsRead →
Clinical trialUNITERAREApr 3

New Recruiting Trial: Study of Silevertinib With Temozolomide for the Treatment of Newly Diagnosed GBM With Unmethylated MGMT and EGFRvIII

Researchers are looking for patients with a specific type of brain cancer called glioblastoma (GBM) to test a new drug combination. The study will test a drug called silevertinib combined with temozolomide in patients whose tumors have certain genetic features (unmethylated MGMT and EGFRvIII). This is an early-stage study to see if this combination works better than current treatments.

WHY IT MATTERSThis trial targets newly diagnosed GBM patients with unmethylated MGMT and EGFRvIII mutations—a subgroup with historically poor prognosis—and offers access to a potentially more effective treatment combination before it becomes widely available.
You can act on thisglioblastomaglioblastoma multiformeRead →
Clinical trialUNITERAREApr 3

New Recruiting Trial: National Surveillance and Prevention of Neonatal VAP

Researchers at the University of Alberta are looking for participants for a study about preventing a serious lung infection called VAP (ventilator-associated pneumonia) in newborn babies. This infection happens when babies on breathing machines in the hospital develop pneumonia. The study will track cases across the country and test ways to stop this infection from happening.

WHY IT MATTERSIf your newborn is in a neonatal intensive care unit (NICU) on a ventilator, this trial could help identify prevention strategies that reduce the risk of developing a serious secondary infection during their hospital stay.
You can act on thisventilator-associated pneumoniaRead →
Clinical trialUNITERAREApr 3

New Recruiting Trial: Comparison of Molecular-Genetic Concordance of the Primary Tumor and Brain Metastases of Gastroesophageal Cancers

Researchers are looking for patients with gastroesophageal cancer (cancer of the food pipe and stomach) that has spread to the brain. This study will compare the genetic makeup of the original tumor with the cancer that spread to the brain to understand if they're the same or different. This information could help doctors choose better treatments for patients whose cancer has spread to the brain.

WHY IT MATTERSIf you have gastroesophageal cancer with brain metastases, this trial could help researchers understand why your cancer spread to the brain and potentially lead to more targeted treatment options in the future.
💬 Ask your doctorgastric canceresophageal cancergastroesophageal cancerRead →
Clinical trialUNITERAREApr 3

New Recruiting Trial: Culturally Adapting an Intervention to Increase Genetic Testing in African American Cancer Survivors

Researchers are looking for African American cancer survivors to join a study about genetic testing. The study will test a new approach designed specifically for African American communities to help more people get tested for inherited cancer genes. Genetic testing can show if someone has genes that increase cancer risk, which helps with prevention and treatment planning.

WHY IT MATTERSThis trial addresses a critical gap: African American cancer survivors are significantly underrepresented in genetic testing programs, meaning many miss opportunities for personalized cancer prevention and family screening.
You can act on thisRead →
Clinical trialUNITERAREApr 3

New Recruiting Trial: MyPEEPS Mobile Plus: A Multi-Level HIV Prevention Intervention for Young MSM

Researchers at Columbia University are testing a new program called MyPEEPS Mobile Plus to help young men who have sex with men prevent HIV infection. The program uses multiple approaches, including mobile technology and community support, to reduce HIV risk. This trial is now accepting participants and will run through 2026.

WHY IT MATTERSThis trial is actively recruiting young MSM (men who have sex with men) ages 18-24 who want to learn evidence-based HIV prevention strategies through a mobile app and peer support program.
You can act on thisRead →
Clinical trialUNITERAREApr 3

New Recruiting Trial: USG-Guided Shoulder Injections in Frozen Shoulder

Researchers are testing a new way to inject medicine into the shoulder using ultrasound guidance to help people with frozen shoulder, a condition where the shoulder becomes stiff and painful. Frozen shoulder makes it hard to move your arm, and this study is looking at whether guided injections can help reduce pain and improve movement. The trial is starting in April 2026 at a hospital in Turkey.

WHY IT MATTERSThis trial offers a potentially more precise injection technique for frozen shoulder patients who haven't found relief with standard treatments, using ultrasound to ensure the medication reaches the right spot in the shoulder joint.
👁 Watch this spaceFrozen ShoulderAdhesive CapsulitisRead →
Clinical trialUNITERAREApr 3

New Recruiting Trial: Evaluation of RBS2418 in Combination With Tremelimumab Plus Durvalumab in Participants With Advanced Unresectable Hepatocellular Carcinoma

Researchers are testing a new combination of three drugs (RBS2418, tremelimumab, and durvalumab) to treat advanced hepatocellular carcinoma, a type of liver cancer that cannot be surgically removed. This is an early-stage clinical trial (Phase 2) that is currently accepting patients. The study aims to see if combining these drugs works better than existing treatments.

WHY IT MATTERSThis trial offers eligible patients with advanced unresectable hepatocellular carcinoma access to a novel three-drug combination that may provide a new treatment option beyond current standard therapies.
💬 Ask your doctorhepatocellular carcinomaRead →
Clinical trialUNITERAREApr 3

New Recruiting Trial: Anthocyanin-Rich Table Grape Powder for Prophylaxis of Post-Operative Atrial Fibrillation

Researchers at the University of Michigan are testing whether a powder made from grapes rich in anthocyanins (natural plant compounds) can help prevent atrial fibrillation—a heart rhythm problem—after heart surgery. This early-stage trial is now accepting patients. The study will test if this natural supplement is safe and if it works better than a placebo (fake treatment).

WHY IT MATTERSThis trial offers patients undergoing heart surgery a potential natural option to reduce their risk of developing irregular heartbeats after the operation, which affects up to 50% of post-operative cardiac patients.
💬 Ask your doctorAtrial FibrillationRead →
Clinical trialUNITERAREApr 3

New Recruiting Trial: A Phase 1 Study of EPI-326 in EGFR-mutant NSCLC and HNSCC

Researchers are testing a new experimental drug called EPI-326 in people with two types of cancer: non-small cell lung cancer and head and neck cancer, both caused by mutations in the EGFR gene. This is an early-stage study (Phase 1) to see if the drug is safe and how much patients can tolerate. The study is currently accepting new participants and is expected to start in April 2026.

WHY IT MATTERSThis trial offers access to a novel EGFR-targeted therapy for patients with EGFR-mutant lung or head and neck cancers who may have limited treatment options or resistance to current therapies.
💬 Ask your doctorEGFR-mutant non-small cell lung cancerEGFR-mutant head and neck squamous cell carcinomaRead →
Clinical trialUNITERAREApr 3

New Recruiting Trial: Evaluation of Xaluritamig in Adults, Adolescents and Children With Relapsed or Refractory Ewing Sarcoma (EWS)

Researchers are testing a new drug called Xaluritamig to treat Ewing sarcoma, a rare bone cancer that has come back or stopped responding to previous treatments. This is an early-stage study (Phase 1) that will include children, teenagers, and adults. The study is now accepting patients and is expected to start in April 2026.

WHY IT MATTERSThis trial offers a potential new treatment option for patients with relapsed or refractory Ewing sarcoma, a rare pediatric cancer with limited treatment alternatives after standard therapies fail.
You can act on thisEwing sarcomaRead →
Clinical trialUNITERAREApr 3

New Recruiting Trial: Phase 3 Study of LUM-201 in Children With Growth Hormone Deficiency

Lumos Pharma is testing a new drug called LUM-201 in children who don't produce enough growth hormone naturally. This Phase 3 trial is the final stage of testing before the drug might be approved by the FDA. Researchers are actively recruiting children to participate and see if this treatment helps them grow taller.

WHY IT MATTERSThis is now actively recruiting children with growth hormone deficiency — families interested in potentially accessing LUM-201 before FDA approval should check eligibility requirements and contact the trial sites listed on ClinicalTrials.gov.
You can act on thisGrowth Hormone DeficiencyRead →
Clinical trialUNITERAREApr 3

New Recruiting Trial: Assessing the Association Between H. Pylori Persistence and the Severity of Insulin Resistance in Patients With Metabolic Syndrome

Researchers are looking for people with metabolic syndrome (a group of conditions including high blood pressure, blood sugar, and cholesterol) to study whether a common stomach bacteria called H. pylori might make insulin resistance worse. Insulin resistance means your body has trouble using a hormone called insulin that controls blood sugar. The study will help doctors understand if treating this bacteria could help people with metabolic syndrome feel better.

WHY IT MATTERSIf H. pylori is found to worsen insulin resistance in metabolic syndrome patients, treating the infection could become a new way to help manage this common condition that affects millions of people.
💬 Ask your doctormetabolic syndromeRead →
Clinical trialUNITERAREApr 3

New Recruiting Trial: Hepzato Kit and Opdualag for Metastatic Melanoma and Liver Metastasis

Researchers at the University of Wisconsin are testing a new combination of two cancer treatments called Hepzato Kit and Opdualag for patients with melanoma (a type of skin cancer) that has spread to the liver. This is an early-stage study that started in 2026 and is currently looking for patients to participate. The goal is to see if this combination can help people whose cancer has spread to multiple parts of their body.

WHY IT MATTERSThis trial offers patients with metastatic melanoma and liver involvement access to a novel combination therapy at a major academic medical center, potentially providing treatment options for a particularly aggressive cancer presentation.
You can act on thismetastatic melanomamelanoma with liver metastasisRead →
← PreviousPage 3 of 5Next →

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