Clinical trialRSSToday
A new experimental drug called tavapadon from AbbVie helped people with early Parkinson's disease delay or avoid starting levodopa, which is the standard treatment for this condition. In a completed Phase 3 trial called TEMPO-4, most people taking tavapadon long-term did not need to start levodopa, and those already taking it didn't need to increase their dose. This suggests tavapadon could be a helpful new option for managing early Parkinson's symptoms.
WHY IT MATTERSIf approved, tavapadon could allow people with early Parkinson's to delay levodopa therapy, potentially postponing side effects that come with long-term use of this standard medication.
Clinical trialRSSYesterday
Scientists tested a new pill called VQ-101 that can enter the brain and increase levels of a protective protein in people with Parkinson's disease. In early testing, the drug worked in patients both with and without a specific genetic change (GBA mutation). This is an important first step toward potentially helping different groups of Parkinson's patients.
WHY IT MATTERSThis is one of the first oral drugs shown to cross the blood-brain barrier and activate neuroprotective pathways in genetically diverse Parkinson's patients, potentially expanding treatment options beyond GBA-mutation carriers.
Clinical trialRSS2 days ago
A company called BlueRock Therapeutics tested a new cell therapy called bemdaneprocel for Parkinson's disease. In a small early-stage trial, patients who received one dose of this therapy showed improvement in their movement symptoms, and these improvements lasted for up to three years. This is promising because it suggests a single treatment might help Parkinson's patients for a long time.
WHY IT MATTERSIf bemdaneprocel continues to show sustained benefits in larger trials, it could offer Parkinson's patients a one-time treatment option instead of daily medications, potentially improving quality of life and treatment adherence.
Clinical trialRSS2 days ago
A new medication called Vyalev helped people with Parkinson's disease stop experiencing unwanted, uncontrolled movements (called dyskinesia) when they switched to it from other Parkinson's drugs. In two clinical trials, most people saw these troublesome movements go away within a few months of starting Vyalev. This is good news because dyskinesia is a common side effect that happens after taking Parkinson's medications for a long time.
WHY IT MATTERSIf you have Parkinson's disease and experience dyskinesia from your current levodopa treatment, switching to Vyalev could eliminate these involuntary movements within months rather than managing them as a permanent side effect.
ResearchRSS2 days ago
Researchers found that a Parkinson's disease medication called Crexont helped patients spend more time feeling better and less time experiencing symptoms when they switched from other levodopa treatments. The study showed improvements in movement control and daily functioning across different patient groups. This is early-stage research being presented at a medical conference.
WHY IT MATTERSIf you take levodopa for Parkinson's and struggle with 'off' periods when symptoms return between doses, Crexont may offer a way to extend your good symptom control time throughout the day.
NewsUNITERARE4 days ago
The PAN Foundation's financial assistance program for phenylketonuria (PKU) — a rare genetic condition where the body can't break down a protein called phenylalanine — is currently closed. This program normally helps patients pay for treatment costs. The fund status changed on April 20, 2026.
WHY IT MATTERSIf you have PKU and were relying on PAN Foundation assistance to afford your medical care or special diet, you need to find alternative financial resources immediately since this program is no longer accepting applications.
Clinical trialUNITERAREApr 15
Researchers are testing a new cancer treatment that combines two approaches: a chemotherapy drug called temozolomide and a vaccine called SurVaxM that trains the immune system to fight cancer cells. This trial is for patients with neuroendocrine carcinomas (rare cancers in hormone-producing cells) that are spreading and getting worse despite other treatments. The study is now accepting patients and will run through 2026.
WHY IT MATTERSThis is one of the first trials testing an immunotherapy vaccine specifically for metastatic neuroendocrine carcinomas, offering a potential new option for patients whose cancer has progressed on standard treatments.
Clinical trialUNITERAREApr 5
Researchers are looking for patients with a rare cancer called adrenocortical carcinoma that has come back after surgery. This study will test whether giving radiation therapy before surgery helps remove the cancer more effectively. The trial is just starting and will enroll patients beginning in April 2026.
WHY IT MATTERSThis is the first Phase 1 trial testing preoperative radiation for recurrent adrenocortical carcinoma, offering eligible patients access to a potentially new treatment approach before it becomes widely available.
Clinical trialUNITERAREApr 5
Researchers are testing a new drug called ManNAc to treat primary focal segmental glomerulosclerosis (FSGS), a rare kidney disease that causes scarring and can lead to kidney failure. This is a Phase 2 trial, meaning the drug has already been tested for safety in a small group and now researchers want to see if it actually works to help patients. The trial is now accepting patients and is expected to start in April 2026.
WHY IT MATTERSThis trial offers patients with primary FSGS access to an investigational treatment that may slow or stop kidney damage, with enrollment now open at sites participating in the National Human Genome Research Institute study.
Clinical trialUNITERAREApr 5
Researchers are testing a new drug called CYT107 to help people with HIV who have Kaposi Sarcoma (a type of cancer) and whose immune systems aren't responding well to treatment. CYT107 is designed to boost the immune system to help fight the cancer. This is a Phase 2 trial, meaning it's testing whether the drug works and is safe in a larger group of people. The trial is now accepting patients and will start in April 2026.
WHY IT MATTERSThis trial offers a potential new treatment option for people with HIV-related Kaposi Sarcoma who haven't responded to standard immune-boosting treatments, addressing a significant gap in care for this vulnerable population.
Clinical trialUNITERAREApr 5
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.
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.
🔴 BreakingDrug approvalOPENFDAApr 4
The FDA has approved a new drug called FOUNDAYO (orforglipron), made by Eli Lilly. This approval means the medication is now available for patients to use. The drug was officially approved on April 1, 2026.
WHY IT MATTERSWithout information about which rare disease FOUNDAYO treats, we cannot specify the patient impact — the article lacks details about the condition it addresses or which patients are eligible.
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 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.
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 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.