Participants will receive a series of scheduled treatment visits where the assigned medication is administered, followed by regular check‑ups that include blood tests and other simple exams to monitor the disease and any side effects. The study lasts for several years, with visits occurring at set intervals, and ends when the participant stops the medication or the study concludes.

The purpose of the study is to see how well Concizumab works in preventing bleeding episodes compared to the previous treatment the children were receiving. The study will involve children who have not used Concizumab before. Participants will receive regular injections of Concizumab over a period of time, and the number of bleeding episodes they experience will be monitored and compared to their previous treatment.

Throughout the study, researchers will also keep track of any side effects or reactions to the medication, such as injection site reactions or the development of antibodies against Concizumab. The study aims to provide valuable information on the safety and effectiveness of Concizumab in managing bleeding episodes in young children with hemophilia.

The purpose of the study is to see whether a higher radiation dose improves overall survival. Participants will be randomly assigned to receive either the standard radiation dose or a higher dose, then all will continue with the same durvalumab treatment. The study follows patients for several months, checking how long they live, how the cancer responds, any side effects, and how they feel in daily life.

The purpose of the study is to determine whether nerandomilast can more effectively delay death or disease worsening compared with the placebo.

Participants will be randomly assigned to receive either the active medication or the placebo for about one year. During this time, they will attend regular clinic visits where doctors will check skin thickness, lung function (including a test called FVC that measures how much air can be forced out of the lungs), physical ability, and overall health. The study will track any serious health events, changes in symptoms, and overall survival throughout the treatment period.

The purpose of the study is to identify the optimal amount of the medication and to evaluate its ability to shrink or stop the growth of the cancer (anti‑tumour activity). The research is conducted as a phase II trial, meaning it is designed to test different dose levels for safety and effectiveness after initial safety has been shown.

Participants receive a series of injections of the study drug into the tumor at scheduled visits, followed by regular medical examinations, imaging scans, and questionnaires that assess symptoms and overall well‑being. The study continues until the treatment plan is completed or until the disease progresses, with ongoing monitoring to record any side effects and changes in the tumor size.

The purpose of the study is to find out which of these treatments best prevents death, heart attack (also called myocardial infarction) or stroke during the first year after the heart event while also keeping serious bleeding low. Participants will take the assigned tablet once daily for up to 12 months and will attend scheduled visits to monitor their health.

During the year, participants will have regular visits where doctors check health, ask about any new problems, and record any occurrences of heart attack, stroke, death, or major bleeding. This information is used to compare the safety and effectiveness of the medicines.

The purpose of the study is to determine whether ALKS 2680 can reduce daytime sleepiness in adults with Narcolepsy Type 1. Participants will be randomly assigned to receive either the study drug or a placebo for about twelve weeks. During the study, they will complete a questionnaire called the Epworth Sleepiness Scale that asks how likely they are to fall asleep in everyday situations, and they will take a short test called the Maintenance of Wakefulness Test that measures how long they can stay awake when asked to do so. Their episodes of cataplexy will also be recorded each week.

Throughout the trial, researchers will monitor participants for any side effects and will collect the questionnaire and test results to evaluate the safety and effectiveness of the medication. The collected information will help decide if the drug provides a beneficial option for managing the symptoms of this sleep disorder.

The main aim of the study is to evaluate how often the tumors shrink, known as an objective response. Participants receive the study drug in treatment cycles that repeat every few weeks, with regular visits for physical exams, blood tests, and imaging scans to check the size of the tumor and overall health.

Tumor shrinkage is classified as a complete response when the cancer disappears, a partial response when the tumor size decreases by a significant amount, or stable disease when the tumor does not grow or shrink much. Throughout the trial, doctors monitor for adverse events, which are side effects or any unwanted medical problems, and record any that are serious or lead to stopping the treatment.

The purpose of the study is to determine whether this therapy improves heart performance and is safe compared with standard care. Participants are randomly assigned to receive either the insulin‑based regimen plus usual treatment or usual treatment alone. The study treatment is given for up to two days while patients remain in the hospital, during which doctors watch heart function, blood pressure, kidney function and any side effects using a thin tube called a catheter (via pulmonary artery catheterization) and regular blood tests. After leaving the hospital, patients are checked again for about six weeks to see if they needed extra heart‑support devices and how well the heart and kidneys are working.

Safety monitoring includes continuous observation of heart rhythm, blood pressure, and the amounts of other heart‑support medicines used. The trial is open label, meaning both the care team and the participants know which treatment is being given, and it is designed as a small pilot to gather early information about the new approach.

The primary aim is to evaluate how well this combination works in controlling the disease. Participants receive the injection and the capsule on a repeated schedule, typically every few weeks, and are followed for several months with regular doctor visits, blood tests, and imaging to check for disease activity and any side effects. The study continues until a predefined number of treatment cycles are completed or if a participant experiences significant toxicity that requires stopping the therapy.

The purpose of this phase II study is to find out whether taking sirolimus after surgery can lead to a lasting reduction in the size of the affected lip and a change in the color of the birthmark, while also checking safety and how the condition impacts quality of life. Participants will start the medication after their surgical procedure and will be followed for several years, with periodic visits to measure the lip’s size with a simple ruler, to assess color changes using a four‑step scale, and to complete questionnaires that ask about daily activities and comfort.

The goal of the trial is to find out whether adding Venetoclax to the standard three‑drug mix can improve overall survival, which means living longer after the treatment starts.

In this phase 3 study, children are placed by chance into one of two groups: one group receives the three standard drugs, and the other group receives the same three drugs plus the oral Venetoclax. Treatment is given in cycles over several weeks, and the children are checked regularly to see how they are doing and to record any side effects. The study follows each participant for a period of time to see how long they live and how they feel.

Healthy volunteers will receive each of the three injections (the two anesthetics and the placebo) in separate study periods, with the order chosen at random and neither the participants nor the staff knowing which injection is given at any time. After each injection, a small device will apply electrical stimulation to create a temporary area of increased pain sensitivity called hyperalgesia, and participants will rate the pain they feel. Additional tests will use a cuff that inflates around the arm to measure the pressure at which pain is first felt and the pressure that can be tolerated, known as cuff-pressure algometry. Throughout the study, blood pressure, heart rate, any side effects and adverse events will be recorded, and each participation period lasts only a few days.

The purpose of the study is to evaluate the safety and how well participants tolerate a step‑up dosing schedule of 25 mg, then 50 mg, and finally 100 mg of the drug. Volunteers will take the tablet at each dose level for a short period, after which they will provide saliva samples to check for the amount of virus present and have blood drawn to measure immune responses such as the number of CD4 T cells that react to the virus using a test called ELISPOT, as well as levels of specific antibodies, including IgG. The study proceeds without complex procedures, allowing participants to continue their normal daily activities while being monitored for any side effects.

The purpose of the study is to see whether oral semaglutide can help slow worsening of memory, thinking, and daily function in people with early Alzheimer’s disease, and to check its safety. The study is randomised, which means the treatment is assigned by chance, and double-blind, which means neither the participants nor the study team knows who receives semaglutide or placebo during the study. Treatment is taken by mouth over a long period, and the study follows changes over time.

Participants take the study tablets regularly and are seen at planned visits during the trial. These visits are used to monitor health, review how the person is doing, and record any changes in memory, daily activities, or side effects. The study compares how people do over time in the semaglutide and placebo groups.

The study uses a step-by-step treatment plan. Each person will have periods with atorvastatin, periods with placebo, and a period with no study treatment. The order of these periods is arranged by chance. During the study, muscle symptoms are checked over time to compare how they change during each period.

The study is designed to help understand statin-associated muscle symptoms, which are muscle problems that some people notice while taking statin medicines such as atorvastatin. It also looks at whether some symptoms may be nocebo or misattributed, meaning they are felt as real symptoms but may not be caused by the medicine itself.

The standard treatment in this study includes bevacizumab, oxaliplatin, fluorouracil, and calcium folinate. INCA33890 is given by vein, and the other medicines are also given as injections or infusions into a vein. People in the study are assigned by chance to one of two groups: one group receives INCA33890 with the standard treatment, and the other group receives placebo with the standard treatment. The study is blinded, which means the treatment group is not known to the people taking part or to the study team during the study.

After treatment starts, the study team follows how the cancer responds and how long the treatment helps keep the disease under control. The study also looks at how long people live and at side effects, which are unwanted health problems caused by a treatment.

The study is planned in two groups, and neither the participants nor the study team will know which treatment is given. After starting treatment, the medicine is taken for a period of time and then the study looks at changes in urine protein and kidney function over about 20 weeks. The study is designed to compare BI 764198 with placebo across the different kidney disease groups.

The study is a Phase 3 trial, which means it is being done in a larger group of people to better understand how well the treatment works and how safe it is. People taking part are assigned by chance to receive either nipocalimab or placebo. The treatment is given over time, and the study team follows participants through the treatment period to observe how the disease changes.

Participants are assigned to one of the study treatments and receive injections over time during regular study visits. The study team follows how the cancer responds and watches for side effects and other health changes. It also looks at how long the treatment effects last and how the treatments affect daily well-being and symptoms.

The study has more than one part. In one part, EIK1005 is given by itself. In another part, EIK1005 is given with pembrolizumab. Participants may take the study tablet on a regular schedule and receive pembrolizumab through a vein during clinic visits. During the study, doctors watch for side effects and other health changes, and treatment may be adjusted as needed. The study also includes people with tumors that are MSI-H or dMMR, which are tumor features linked to problems in how cancer cells repair damage in their genetic material.

EIK1005 is also called a Werner helicase inhibitor. A helicase is a protein that helps cells handle and copy genetic material, and this medicine is designed to block that protein. The trial is planned to continue over several years while safety and dose information are collected.

People in the study are placed into one of the treatment groups by chance. Treatment is given as intravenous infusion, which means medicine is delivered slowly through a vein. The study is planned to follow people over time while they receive treatment and after treatment ends to see how they do. The trial also watches for side effects, including cytokine release syndrome, a strong immune reaction, and ICANS, which is a group of brain and nerve symptoms that can happen with some immune treatments.

Obrixtamig is also known by the code name BI 764532. It is a type of treatment called a T cell engager, which is designed to help the immune system find and attack cancer cells that have DLL3 on their surface. The study is for previously untreated cancer that is DLL3-positive, meaning the cancer cells have this marker.

The study lasts about 24 weeks. Participants are assigned by chance to receive either lerodalcibep or placebo, and neither the family nor the study team knows which one is given during the study. The medicine is given once a month, and study visits are spread over the treatment period. Doctors also check general health, growth, and development, and watch for side effects and other safety concerns.

The main purpose of the study is to find out whether lerodalcibep is safe and effective for lowering LDL-C in children and adolescents with this inherited cholesterol disorder. Other blood fats and some body changes related to growth and hormones are also followed during the study.

People in the study receive ifinatamab deruxtecan over time during regular treatment visits. The study is open-label, which means both the study team and the participant know which treatment is being given. The course of the study includes treatment and follow-up visits to watch for side effects and to see how long any benefit lasts. The study also looks at how long people live without the cancer getting worse and how many side effects happen during treatment.

In the study, people are randomly assigned to receive either the new treatment combination or the standard treatment combination. The medicines are given as intravenous infusion, which means they are delivered slowly through a vein. Treatment is given in cycles over time, with regular visits for infusions and checks by the study team. The study will look at how long people live and will also follow symptoms and side effects, including breathing problems, chest pain, cough, and treatment-related reactions such as CRS and ICANS. CRS, or cytokine release syndrome, is a strong immune reaction that can cause fever and other symptoms. ICANS, or immune effector cell-associated neurotoxicity syndrome, is a brain and nerve problem that can affect thinking, speech, or alertness.

The purpose of the study is to compare the effectiveness of a new medicine with that of standard chemotherapy. The investigational drug being tested is Sofetabart Mipitecan (LY4170156). For participants with platinum-resistant disease, the new drug may be given alone and compared with the doctor’s choice of chemotherapy drugs such as carboplatin, paclitaxel, gemcitabine, topotecan or doxorubicin, or with Mirvetuximab Soravtansine. For those with platinum-sensitive disease, the new drug is combined with bevacizumab and compared with a standard platinum‑based two‑drug chemotherapy regimen plus bevacizumab.

Participants receive the study medicines through an IV infusion every few weeks and attend regular clinic visits where doctors perform physical examinations and imaging scans to check how the cancer is responding. Treatment continues until the cancer grows, side effects become unacceptable, or the study period ends, which may be several months for each participant.

People in the study are assigned by chance to receive either AZD0120 or one of the standard treatment plans used for this disease. The standard plans may include combinations such as daratumumab, carfilzomib, bortezomib, pomalidomide, and dexamethasone. AZD0120 is given through a vein, while the other treatments may be given by infusion, injection, or as capsules or tablets taken by mouth. The study then follows participants over time to see how the disease changes, how long the treatment effects last, and how safe the treatments are, with regular checks of general health and blood tests.

Participants in the study will receive medications through an intravenous infusion, which is a method of delivering medicine directly into a vein. The study involves comparing different combinations of drugs to see how they affect progression-free survival, a term used to describe the length of time during and after treatment that a person lives with the disease without it getting worse. Other factors being observed include overall survival and the quality of life, which tracks how well a person can perform daily activities and their general well-being during the study.

The purpose of the study is to compare the effectiveness and safety of the two treatments in reducing the need for red blood cell transfusions. Participants are randomly assigned to receive either elritercept or epoetin alfa and will be treated for up to 24 weeks. During this time, they will have regular clinic visits where blood samples are taken to check hemoglobin levels, which indicate how well the blood is carrying oxygen, and doctors will record whether transfusions are still needed.

The study involves a series of visits scheduled roughly every few weeks. At each visit, a healthcare professional will administer the injection, perform a quick blood test, and ask about any side effects. The overall goal is to see if participants can stay without transfusions for a sustained period while their hemoglobin improves.

The aim of the study is to evaluate the long‑term safety of the medicine and to see if it can help increase height. Participants will take the capsule each day and attend regular visits where doctors check vital signs, perform blood tests, eye and dental exams, and take simple scans such as X‑rays and a bone‑density scan called dual x‑ray absorptiometry to monitor growth. Height will be measured over time and compared with standard growth charts.

The purpose of the study is to evaluate the safety and effectiveness of AAVB-039. The investigation is divided into two parts. The first part focuses on finding the safest and most appropriate amount of the medicine to use. The second part examines how well the treatment works at that specific dose over time.

Participants in the study will be monitored to observe any changes in their vision and the health of their eyes. This includes checking the ellipsoid zone, which is a specific layer of cells in the retina that is important for clear vision, and the macula, the part of the eye responsible for detailed central sight. The study will track these changes over several years to understand the long-term impact of the treatment.

Participants will receive the medication to see how it affects the number of days they experience a migraine each month. During the study, the frequency, intensity, and duration of the headaches will be monitored. The researchers will also observe any adverse events, which are any side effects or unexpected health changes that occur during the treatment period.

In this study, participants will receive either rinatabart sesutecan or a treatment selected by their doctor, which may include medications such as doxorubicin or paclitaxel. These medications are administered through an IV infusion, a method where medicine is delivered directly into a vein through a small tube. The study aims to determine if the new drug works better than the standard options currently available for this condition.

During the course of the study, participants will undergo regular monitoring to track how the cancer responds to the medication and to observe any side effects. The researchers will look at how long the cancer remains stable without growing and the overall survival of the participants. This process involves continuous observation over several years to collect data on how the treatments affect the health and well-being of the individuals involved.

The purpose of the study is to determine if BI 764198 is more effective than a placebo at reducing the amount of protein leaked into the urine. During the study, participants will take either the active medication or a placebo in the form of an oral film-coated tablet. The treatment period lasts for 104 weeks, and the process is double-blind, meaning neither the participants nor the researchers know which treatment is being administered during the trial.

The placebo used in this study consists of sodium chloride. To evaluate how well the treatments work, the size of the injured area in the brain will be measured using MRI, a medical imaging technique that uses magnets to create detailed pictures of the body. Specifically, the study looks at the growth of the area of dead brain tissue over a period of 72 hours. Other aspects of the study involve monitoring physical health, potential side effects, and changes in certain substances in the blood that relate to inflammation.

Participants will be assigned to receive either olpasiran or a placebo through a subcutaneous injection, which means the medication is delivered into the fatty tissue just under the skin. During the study, researchers will monitor for various health events such as ischemic stroke, which is a blockage of blood flow to the brain, and cardiovascular death. The study will also track changes in the levels of lipoprotein (a) in the blood over time to see how the treatment affects this substance.

Participants in this trial will receive either the study drug, remibrutinib, in the form of a film-coated tablet taken by mouth, or a placebo. The purpose of the study is to determine if this treatment can help delay the progression of disability. During the study, individuals will be monitored over a period of time to observe how the medication affects their physical abilities and overall health.

Participants in the study will be assigned to receive different combinations of medications. One group will receive Etentamig, which is given as an intravenous infusion, alongside Daratumumab. The other group will receive a combination of Daratumumab, Lenalidomide, and Dexamethasone. Dexamethasone is a type of steroid used to reduce inflammation, and Lenalidomide is an oral medication often used to treat cancer.

The study is divided into two main parts. The first part aims to find the correct amount of Etentamig to use when combined with Daratumumab. The second part compares the effectiveness of the Etentamig and Daratumumab combination against the standard treatment of Daratumumab, Lenalidomide, and Dexamethasone. During the study, participants will be monitored to see how the treatments affect the disease and to track any side effects.

The purpose of this study is to find out whether the study medications are better than placebo at reducing the occurrence of major liver-related complications. During the study, researchers will monitor participants for various liver problems including progression to cirrhosis (severe liver scarring), development of complications like varices (enlarged veins in the digestive tract), ascites (fluid buildup in the abdomen), hepatic encephalopathy (brain function problems caused by liver disease), and other serious liver conditions. The study will also track whether participants need liver transplantation or experience other serious outcomes.

Participants in this study will receive their assigned treatment over an extended period while being regularly monitored by the study team. The study requires participants to have evidence of liver fat confirmed by MRI (a type of imaging scan) and liver scarring confirmed through non-invasive tests, though a liver biopsy is not required to join the study. Throughout the study, participants will have regular check-ups and assessments to evaluate how well the treatment is working and to monitor their liver health and overall wellbeing.

The purpose of this study is to evaluate how safe and tolerable the combination of MK-2870 and chemotherapy is for patients. The study will also look at how well the treatment works in shrinking or controlling the cancer. Participants will receive the study medications through an intravenous infusion, which means the drugs are given directly into a vein. The study will monitor participants for any side effects and measure how the cancer responds to the treatment over time.

In addition to MK-2870 and paclitaxel, the study may involve other medications such as buclizine hydrochloride, paracetamol (also known as acetaminophen), codeine phosphate, ramucirumab, and glucocorticoids. These medications may be used to manage symptoms or side effects. The study aims to provide valuable information on the potential benefits and risks of these combination therapies for patients with advanced gastroesophageal cancer.