Participants who join the extension will continue to take the granules for oral solution each day, with regular clinic visits to check for any side effects and to perform simple functional tests. These tests include the NSAD, a questionnaire that rates everyday activities such as walking and climbing stairs; the 10MWT, which measures how fast a person can walk ten meters; the FVC, a breathing test that estimates lung capacity while seated; the PUL 2.0, an assessment of arm and hand function for those who cannot walk; the 100MTT, a longer walking test; and a blood draw to measure serum CK, an enzyme that rises when muscle tissue is damaged. The schedule is designed to monitor health over an extended period without requiring invasive procedures.

Participants will start the medication and, when appropriate, undergo surgery and laser sessions. Over a period of up to five years, they will have regular check‑ups where doctors measure the three‑dimensional size of the lip with a simple caliper, rate the color of the birthmark into four categories (light red/pink, deep red, bluish red, or purple), and complete a short questionnaire about how the condition affects daily life. A port‑wine stain is a reddish birthmark caused by abnormal blood vessels; macrocheilia simply means a larger‑than‑normal lip; laser therapy uses focused light to improve skin appearance; and quality of life reflects a person’s overall well‑being and ability to carry out everyday activities.

This is a phase II, open‑label trial, meaning all participants receive the active drugs and both investigators and participants know which treatment is given. Participants receive the injection and the pill on a regular schedule for up to 12 treatment cycles, roughly one year, with additional follow‑up visits after treatment ends. Throughout the study, doctors check for signs that the disease is not getting worse, measured as progression‑free survival, and look for a complete response, which means no detectable disease. They also record overall length of survival (overall survival) and monitor any adverse events, or side effects, to assess safety.

The standard chemotherapy regimen includes three medicines: fludarabine, which is given by injection under the skin; cytarabine, a drug given through a vein at a high dose; and gemtuzumab ozogamicin, an antibody‑linked drug also delivered by IV. Participants are randomly assigned to receive either this combination alone or the same combination together with the oral drug venetoclax, taken each day in a liquid form.

After the initial treatment cycles, children will have regular check‑ups to monitor their health and to record how long they live, without requiring any additional experimental procedures. The study follows each child for several months to years to collect survival data and to observe any side effects.

The purpose of the study is to find out whether the medication can slow the narrowing of the valve and improve the ability to exercise. Participants are randomly assigned to receive either the study drug or the placebo for about a year, with regular clinic visits. During the study, doctors will use an echocardiogram (an ultrasound picture of the heart) to see how big the valve opening is, and a peak VO2 test, measured during a CPET (a breathing and heart test done while exercising), to check how well the body uses oxygen. A CT scan (a detailed X‑ray picture) will also be performed to look at calcium buildup in the valve. If the valve becomes too narrow, doctors may discuss procedures such as TAVR (a catheter‑based valve replacement) or SAVR (surgical valve replacement).

Throughout the trial, participants will be monitored for any side effects, and safety checks will be done at each visit. The study team will collect information about health changes and any additional treatments needed, while keeping participants’ personal information confidential.

The purpose of the study is to assess how effective and safe sonelokimab is for adults with moderate to severe hidradenitis suppurativa. Participants in the study will receive either sonelokimab or a placebo over a period of 16 weeks. During this time, researchers will monitor the participants to see if there is a 75% improvement in their condition, which is measured by a specific score called the Hidradenitis Suppurativa Clinical Response (HiSCR) score. The study will also track any side effects or adverse events that occur during the treatment period.

Throughout the study, participants will undergo regular check-ups, including physical examinations and laboratory tests, to ensure their safety and to gather data on the treatment’s effects. The trial aims to provide valuable information on whether sonelokimab can be a beneficial treatment option for those suffering from hidradenitis suppurativa, potentially improving their quality of life by reducing the severity of their symptoms.

The purpose of the study is to see how well Nemtabrutinib works compared to the other two treatments in people who have not yet received treatment for their CLL or SLL. Participants will be randomly assigned to receive either Nemtabrutinib or one of the other two medications chosen by the study doctor. The study will last for a period of up to 108 weeks, during which participants will take the medication daily and attend regular check-ups to monitor their health and the progress of the disease.

Throughout the study, doctors will assess how the disease responds to the treatment and how long participants remain free from disease progression. They will also monitor overall survival and any side effects experienced by participants. The study aims to provide valuable information on the effectiveness and safety of Nemtabrutinib compared to the current standard treatments for CLL and SLL.

Participants in the study will be randomly assigned to receive either atorvastatin or a placebo, which is a tablet that looks like the medication but does not contain the active ingredient. The study will compare the effects of two different doses of atorvastatin, 20 mg and 40 mg, to see if they can decrease the frequency of migraines. The trial will last for several weeks, during which participants will take the medication daily and report the number of migraine days they experience.

The purpose of this study is to confirm whether the positive effects of atorvastatin observed in smaller studies can be replicated in a larger group of people. By participating, individuals will help researchers understand if atorvastatin can be an effective preventative treatment for those suffering from episodic migraines. The study is designed to be thorough and will involve multiple centers to ensure a comprehensive evaluation of the medication’s effects.

The main goal of the study is to see if the medication is safe and how well it reaches the CNS and influences a brain chemical known as 4-hydroxy-2-nonenal. Over a period of about 12 weeks, participants will take the assigned pill daily and attend regular check‑ups. At the beginning and end of the study, a small amount of fluid surrounding the brain and spinal cord, called CSF, may be collected through a thin needle to measure the level of the drug’s breakdown product AD-835 and the amount of the brain chemical. Simple brain scans using 31P-MRS may be performed to assess the brain’s energy use, and blood samples will be analyzed with LC‑MS. Laboratory tests such as ELISA, TBARS, and measurement of MDA will be used to evaluate the brain chemical levels.

The purpose of the study is to assess the effectiveness and safety of OATD-01 over a 12-week period. Participants will be randomly assigned to receive either OATD-01 or a placebo, and neither the participants nor the researchers will know which treatment is being administered. Throughout the study, participants will undergo various assessments, including imaging tests like [18F]FDG PET/CT, to monitor changes in lung inflammation and overall health. The study aims to determine if OATD-01 can reduce the inflammation in the lungs and improve lung function and quality of life for those with active pulmonary sarcoidosis.

Participants will be closely monitored for any side effects or changes in their health during the study. The trial will also collect data on various health parameters, such as lung function tests and quality of life questionnaires, to evaluate the overall impact of the treatment. The study is expected to provide valuable insights into the potential benefits of OATD-01 for individuals with active pulmonary sarcoidosis.

Participants are randomly assigned to either the active drug or placebo and will take the tablets daily for roughly 18 months alongside their cancer treatment. Heart function is measured at the start and at the end of the study using CMR, a special type of MRI that visualizes the heart. The primary assessment is the change in LVEF, which indicates how well the left side of the heart pumps blood. Additional evaluations include GLS, a measure of how the heart muscle stretches during beats, and blood tests for NT-proBNP and the high‑sensitivity cardiac injury markers hs‑TnI and hs‑TnT.

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 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.

The purpose of the study is to determine if niraparib can help patients live longer without the disease getting worse, compared to temozolomide. Participants in the study will be randomly assigned to receive either niraparib or temozolomide. The study will monitor the participants over time to see how the treatments affect their health and the progression of their glioblastoma.

Throughout the study, researchers will also look at how the treatments impact overall survival, which means the length of time patients live after starting the treatment. Additionally, the study will assess the participants’ symptoms, quality of life, and any side effects they may experience. This information will help determine the safety and effectiveness of niraparib compared to temozolomide for treating this specific type of glioblastoma.

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 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.

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 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.

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.

People in the study are assigned to one of the two medicines. The treatment is given over time as intravenous infusions, which means medicine is put directly into a vein. The study is double-blind, which means the people taking part and the study doctors do not know which treatment is being given. During the study, doctors follow how the cancer changes, watch for side effects, and check how well each medicine is tolerated.

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.

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.

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.

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.

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.

Children are randomly assigned to receive either the study drug or the placebo during the operation. The anesthesia team uses heart rate variability and electroencephalography to guide how deep the anesthesia is. After surgery, the child’s behavior is checked at 15, 30 and 45 minutes after waking using the PAEDs score and simple pain scales to see if delirium or pain is present. The length of stay in the recovery area and any side effects are also recorded.

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 study is designed as a split-body study, which means that different treatments will be applied to different areas of the body on the same person. Before starting the maintenance treatment with the creams, patients will first receive treatment with a corticosteroid cream, which is a medication that reduces inflammation, for about four weeks to clear the skin lesions. Corticosteroids are commonly used to treat skin inflammation and help calm down active eczema flare-ups. Once the skin has cleared, the maintenance phase begins, where one moisturizing cream will be applied to one study area on the body, another cream or no treatment will be applied to another area, and this will continue for up to 90 days or until the eczema comes back.

During the study, the time it takes for the eczema to return on each treated area will be measured, which is the main focus of the research. Other aspects that will be looked at include how itching changes over time in the different areas and whether any unwanted effects occur during the treatment period. Patients will report when they notice their eczema returning and how much itching they experience using a rating scale.