ResearchBIORXIVApr 5
Researchers studied the genes of people with pulmonary fibrosis (a disease where lung tissue becomes scarred and stiff) to find rare genetic changes that might cause the disease. They found that problems in genes related to how cells maintain their structure may play an important role in developing pulmonary fibrosis. This discovery could help doctors better understand why some people develop this serious lung condition.
WHY IT MATTERSIf you or a family member has pulmonary fibrosis, this research could eventually help explain your genetic risk and lead to new treatments targeting the cellular structures that are damaged in this disease.
Clinical trialUNITERAREApr 4
Researchers at Dartmouth-Hitchcock Medical Center are testing a therapy called TF-CBT (Trauma-Focused Cognitive Behavioral Therapy) to see if it works well for autistic young people. This is a pilot study, which means it's a small test to see if the approach is worth studying more carefully. The study is now accepting participants and is expected to start in April 2026.
WHY IT MATTERSThis trial addresses a significant gap in mental health treatment for autistic youth, as trauma-focused therapies are often adapted without testing whether they work safely and effectively for autistic individuals.
Clinical trialUNITERAREApr 4
Researchers are testing a new drug called JL15003 to see if it can safely treat glioblastoma, a serious brain cancer that has come back after previous treatment. This is an early-stage study that will check both how safe the drug is and whether it helps patients live longer or feel better. The study is now accepting patients and will run starting in April 2026.
WHY IT MATTERSThis trial offers a potential new treatment option for patients with recurrent glioblastoma, a condition with very limited treatment choices after initial therapy fails.
ResearchBIORXIVApr 4
Researchers developed a new test called targeted long-read sequencing that can better identify which genetic changes affect how genes are read and used in cells. This test is more accurate than older methods at finding these changes in patients with neurological disorders, and it's faster and cheaper than previous approaches.
WHY IT MATTERSIf you have a neurological disorder where genetic testing hasn't found a clear diagnosis, this method could help doctors identify the actual genetic cause by better detecting splicing variants that standard tests miss.
Clinical trialUNITERAREApr 3
Researchers at the University of Alabama at Birmingham are looking for premature babies to join a study about pulmonary hypertension—a condition where blood pressure in the lungs gets too high. The study will test different oxygen level targets to see which approach helps prevent or reduce lung problems in preterm infants. This trial is now actively recruiting participants and is expected to start in April 2026.
WHY IT MATTERSThis trial directly addresses a common complication in premature infants where improper oxygen management can cause lasting lung damage, making oxygen saturation targeting strategies critical for improving long-term outcomes in this vulnerable population.
ResearchUNITERAREApr 3
This is a research study looking for patients and families affected by rare diseases to share their thoughts and values about brain and nerve-related medical decisions. Researchers at St. Jude Children's Research Hospital want to understand what matters most to people living with rare diseases so they can make better ethical decisions in medical research and treatment. The study is now accepting participants and will start in April 2026.
WHY IT MATTERSThis study gives rare disease patients and caregivers a direct voice in shaping how medical ethics and brain-related research decisions are made — your input could influence future treatment guidelines and research practices.
Clinical trialUNITERAREApr 3
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.
Clinical trialUNITERAREApr 3
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.
Clinical trialCLINICALTRIALSApr 3
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.
Clinical trialCLINICALTRIALSApr 3
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.
Clinical trialUNITERAREApr 3
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.
Clinical trialUNITERAREApr 3
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.
Clinical trialUNITERAREApr 3
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.
ResearchPUBMEDApr 3
This research review looked at how mental health problems and rare diseases are connected, especially during the long process of getting diagnosed. Researchers found four main themes: patients struggling with hope and hopelessness, confusion about their identity, feeling alone or connected to others, and difficulty accessing mental health services that understand rare diseases. The study shows that mental health care and rare disease care need to work better together.
WHY IT MATTERSIf you or a loved one has a rare disease, this research validates that the emotional and mental health challenges you face during diagnosis and treatment are real and documented — and highlights why doctors should screen for depression and anxiety as part of rare disease care.
Clinical trialUNITERAREApr 3
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.
Clinical trialUNITERAREApr 3
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.
Clinical trialUNITERAREApr 3
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.
Clinical trialUNITERAREApr 3
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.
Clinical trialUNITERAREApr 3
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.
Clinical trialUNITERAREApr 3
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.