Clinical trialRSSYesterday
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 trialRSS2 days ago
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 trialRSS3 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.
NewsUNITERARE5 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 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 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.
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 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.
Clinical trialUNITERAREApr 3
Researchers are looking for patients with a type of blood cancer called acute lymphoblastic leukemia (ALL) to test a new treatment combination. The study will test whether adding a drug called JZP458 to standard chemotherapy works better than chemotherapy alone. This trial is just starting and will recruit patients beginning in April 2026.
WHY IT MATTERSThis Phase 2 trial is now actively recruiting newly diagnosed ALL patients without the Philadelphia chromosome, offering access to an investigational asparaginase formulation (JZP458) that may have improved tolerability compared to standard asparaginase.
Clinical trialUNITERAREApr 3
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.
NewsUNITERAREApr 3
The PAN Foundation's financial assistance program for phenylketonuria (PKU) — a rare genetic condition that affects how the body processes a protein called phenylalanine — is currently closed and not accepting new applications. The program previously offered up to $12,000 per year to help patients pay for treatment costs. The fund status changed on April 2, 2026.
WHY IT MATTERSIf you have PKU and were relying on this $12,000 annual assistance to afford your special diet or medications, you'll need to find alternative financial support resources immediately.
NewsUNITERAREApr 3
The PAN Foundation's financial assistance fund for ALS (a disease that affects nerve cells controlling muscles) is currently closed and not accepting new applications. This fund previously provided up to $12,000 per year to help patients pay for treatment costs. The fund status changed on April 2, 2026.
WHY IT MATTERSIf you have ALS and were counting on this $12,000 annual assistance from PAN Foundation, you'll need to explore other financial resources immediately, as this fund is no longer available.
NewsUNITERAREApr 3
The PAN Foundation's financial assistance program for Duchenne muscular dystrophy patients has closed as of April 2, 2026. This program previously provided up to $12,000 per year to help eligible patients pay for treatment costs. Patients who need financial help with their DMD care should contact PAN Foundation or explore other assistance programs.
WHY IT MATTERSPatients with Duchenne muscular dystrophy who were relying on this $12,000 annual benefit to afford medications or treatments now need to find alternative financial assistance sources immediately.
NewsUNITERAREApr 3
The PAN Foundation's financial assistance fund for paroxysmal nocturnal hemoglobinuria (PNH) — a rare blood disorder where red blood cells break down too quickly — is currently closed and stopped accepting new applications as of April 2, 2026. This fund previously provided up to $12,000 per year to help patients pay for treatment costs. Patients who need financial help with their PNH medications should check the PAN Foundation website for updates on when the fund might reopen.
WHY IT MATTERSPatients with PNH who were relying on this $12,000 annual benefit to afford their medications now need to find alternative financial assistance programs immediately, as this major funding source is no longer available.