The purpose of the study is to find out if the medicine can reduce skin thickening. Participants will receive the assigned treatment every few weeks for about six months, with regular visits to check safety and to measure skin changes using the modified Rodnan skin score, a simple scale doctors use to rate how tight the skin feels. Small blood samples and occasional skin samples may be taken to look for any reactions, and any side effects will be recorded throughout the study.

The purpose of the study is to assess the safety and tolerability of DNL952 in adults with Late-Onset Pompe Disease. Participants will receive the infusion at several clinic visits and will be checked for any side effects. Small blood samples will be taken to understand the drug’s pharmacokinetics (how the body absorbs, moves, and clears the medicine) and pharmacodynamics (how the medicine works in the body). The study will also look for the development of ADAs, which are antibodies the body might produce against the drug. The overall time in the study is a few months, with regular monitoring to ensure participant safety.

In the study, treatment is given over a long period of time, and people are followed while they receive one of the study medicines. The study looks at how often a blood clot in a vein happens after treatment starts. The medicines being studied are baricitinib, adalimumab, and etanercept.

The trial will compare the effects of Clazakizumab with a placebo, which is a substance that looks like the medication but does not contain the active ingredient. The study will also use a saline solution, which is a simple saltwater solution, as part of the trial process. The purpose of the study is to determine the best dose of Clazakizumab that can effectively reduce inflammation and to assess its safety in patients with ESKD. Participants will receive the medication or placebo through an intravenous injection, which means it is given directly into a vein.

Throughout the study, researchers will monitor the participants’ health and track any changes in their condition. The study will also look at the impact of Clazakizumab on cardiovascular health, which refers to the health of the heart and blood vessels. This is important because patients with ESKD are at a higher risk of developing heart-related issues. The trial is designed to provide valuable information on how Clazakizumab can help manage inflammation and improve overall health outcomes for patients with ESKD undergoing dialysis.

Participants are randomly assigned to receive either the lower or the standard dose of the albumin infusion at the time of the fluid‑removal procedure and are then followed for up to one year. During this period, they will have regular clinic visits where blood tests and simple questionnaires are used to check for any new health issues, such as infections of the abdominal fluid (spontaneous bacterial peritonitis), kidney problems caused by liver disease (hepatorenal syndrome – acute kidney injury), bleeding from enlarged veins in the esophagus (variceal bleeding), or changes in brain function due to liver failure (hepatic encephalopathy). The study also records overall well‑being and any hospital visits, but no technical details of the measurements are provided.

The purpose of the study is to assess whether SAR445399 can lower the amount of mucus plugs in the lungs and improve overall lung health compared with placebo. The main measurement is the change in mucus plug score obtained from high-resolution computerized tomography, a detailed chest scan that shows the presence of mucus blockages. Additional evaluations include changes in forced expiratory volume in 1 second, a test that measures how much air can be expelled in one second, and the frequency of pulmonary exacerbation, which refers to episodes of worsening breathing problems that may require extra treatment.

Participants will be randomly assigned to one of the two groups and will receive the assigned injection at regular clinic visits over a period of several months. Throughout the study, participants will undergo the chest scan and breathing tests at the start and at later visits, and their safety will be monitored through routine check‑ups and laboratory tests.

The study aims to find out whether it is possible to run a larger trial that looks at the added benefit of this extra oxygen treatment. Participants are randomly assigned to either receive the supplemental oxygen sessions or to receive the usual care without the extra oxygen. The trial follows each person for several weeks after surgery, recording how many oxygen sessions are completed and collecting information on recovery and how they feel.

The main goal of the trial is to see whether AGA2115 can increase bone mineral density (BMD) in the lumbar spine after one year of treatment. Adults with the condition will be randomly assigned to either the active drug or the placebo group and will be followed for about 12 months, with regular visits to check safety and measure bone density. The study will collect information on any side effects and overall health while participants continue their usual care.

The purpose is to evaluate the safety and tolerability of single doses of the study drug. After receiving one injection, participants are monitored for several weeks to track any side effects and to assess how the drug moves through the body (pharmacokinetics) and how it influences bodily functions (pharmacodynamics). The trial uses a randomized, double‑blind design, meaning neither the participants nor the study staff know which injection is the active drug or the placebo, and it includes a short series of visits for screening, dosing, and follow‑up assessments.

During the study, volunteers will attend regular clinic visits over about 12 weeks. At each visit, doctors will check how the disease is doing using a scoring system called the Modified Mayo Score, which looks at symptoms, a visual exam of the colon (called an endoscopic exam), and lab results. A “clinical remission” means the person’s symptoms have become minimal or disappeared, while a “clinical response” means the symptoms have improved but may not be completely gone. An “endoscopic improvement” indicates that the lining of the colon looks better when viewed with a camera, and “histologic” improvement means that tissue samples show less inflammation under a microscope. Safety will be monitored by recording any side effects and noting if anyone stops the treatment because of them.

The purpose of the study is to evaluate whether Orelabrutinib can delay the worsening of disability compared with placebo. Participants will receive the assigned tablet each day for several years and will attend regular clinic visits where their ability to perform everyday tasks is checked and brain scans using MRI are performed to look for new lesions. The study will track how long it takes before a confirmed increase in disability occurs and will record any safety concerns throughout the trial.

The main aim is to find out whether this combined approach is safe, practical, and helpful for veterans whose symptoms have not improved with other treatments.

During the trial, participants will attend several visits over a few months. After an initial screening, they will receive a low dose of the medication followed by a higher dose in separate sessions, each accompanied by a therapist. Between sessions, they will have regular follow‑up appointments to monitor health, discuss experiences, and complete questionnaires about symptoms, mood, and daily functioning.

The purpose of the study is to watch for any long‑term safety concerns, specifically the chance of developing malignancy caused by insertional oncogenesis, a process where the therapy’s genetic material might unintentionally trigger cancer. After receiving the infusion, participants will have regular sample collections over many years so doctors can check for signs of cancer or unusually high levels of altered cells. This monitoring helps ensure that any potential risks are identified early while the treatment’s benefits continue to be evaluated.

The purpose of the study is to evaluate how effective and safe fidrisertib is for both children and adults with FOP. Participants in the study will take the medication in the form of a hard capsule by mouth. The study will last for a period of up to 60 days, during which participants will be monitored for any changes in their condition and any side effects they might experience. The study will use imaging techniques like computed tomography (CT) to measure changes in bone formation.

Throughout the study, participants will have regular check-ups to assess their health and the progress of their condition. The study aims to provide valuable information on whether fidrisertib can effectively slow down or stop the progression of FOP, offering hope for better management of this challenging condition. Participants will be closely monitored to ensure their safety and well-being during the trial.

The purpose of the study is to determine whether this dose of the drug reduces the likelihood of developing pancreatitis after the procedure. Participants receive the infusion shortly before the ERCP, remain in the hospital for observation, and are checked for abdominal pain, blood test results, and any imaging signs of pancreatitis for up to 48 hours, with additional follow‑up for about a month to monitor any complications or side effects.

After the initial monitoring period, participants may have brief clinic visits or phone calls to assess recovery, quality of life, and any additional medical care needed. A small number of participants will also have blood drawn to measure drug levels, helping researchers understand how the medicine behaves in the body.

The purpose of the study is to determine whether Admilparant can be used safely over an extended period without causing serious side effects. Participants will take the medication each day and will attend regular visits where healthcare staff will check vital signs such as blood pressure and heart rate, review simple blood tests, and perform a basic heart test known as an ECG, which records the heart’s electrical activity. Any side effects that arise will be recorded and evaluated.

The study will continue for many months to gather enough information about safety and tolerability. Throughout the study, participants will be asked to report any new symptoms or problems, and they will receive routine medical check‑ups to monitor their overall health while using the medication.

The main goal of the trial is to evaluate the long‑term safety and tolerability of the study drug in people with the condition. After an initial screening, participants receive the medication at regular intervals for an extended period, during which doctors monitor vital signs, blood tests, heart recordings, and physical examinations. Simple walking and climbing tests are performed at the start and at later visits to see how the treatment may affect mobility.

Throughout the study, blood samples are taken to measure how much of the medicine remains in the body and to check for any immune response. All observations are recorded to help determine whether the therapy is safe for continued use over time.

Participants receive three cycles of the infusion, each given several weeks apart, and then are monitored for signs that the disease returns or worsens. Follow‑up includes regular scans and doctor visits to check for any new growths, side effects, or overall health changes. The study records the time until any cancer recurrence, any death related to the disease, and any serious side effects, using standard criteria such as RECIST 1.1 to measure tumor changes.

After the surgery, each person receives the assigned injection—either under the skin (subcutaneous) or into a muscle (intramuscular)—on a schedule set by the study team. Follow‑up visits are planned regularly to check health, monitor any side effects, and assess overall well‑being.

During the study, doctors will watch for any adverse events (unwanted health problems) and ask participants to complete questionnaires about their quality of life and daily functioning. This information helps determine if the treatment is safe and if it improves outcomes compared with the control injection.

Participants receive the drug in a stepwise manner, starting with low doses that are gradually increased to find a level that can be tolerated without serious side effects. Once a safe dose is identified, additional participants receive that dose to see whether the cancer shows signs of shrinking or slowing. Throughout the study, regular visits include physical examinations, blood tests that measure substances such as PSA (a protein that can indicate prostate cancer activity), and imaging scans that create pictures of the inside of the body. Any side effects, changes in laboratory results, or new symptoms are recorded, and the drug may be stopped if safety concerns arise.

The main aim of the study is to see whether this new regimen works better and is safe compared with the usual care for these cancers.

Participants will receive the study medicines in repeated treatment cycles, each lasting a few weeks, with regular check‑ups to monitor tumor size and overall health. Tumor changes are measured using standard imaging and a set of rules called RECIST 1.1. The study looks at how long patients live without the cancer getting worse, known as PFS, and how long patients live overall, referred to as OS. Blood tests and heart checks are done throughout to watch for side effects.

Participants in the study will take lasmiditan as needed for their migraine attacks over a 12-month period. The study will monitor how well the medication is tolerated by the participants and will track any side effects that may occur. The study will also look at how many participants stop using the medication due to any adverse effects.

Throughout the study, participants will be closely observed to ensure their safety and to gather information on how lasmiditan works in treating migraines in children. This research aims to provide valuable insights into the long-term use of lasmiditan for managing migraines in pediatric patients.

The purpose of the study is to assess the long‑term safety and clinical benefit of the medication. Participants will take the granules by mouth each day for an extended period and will attend regular visits where doctors will check how they are feeling and perform simple tests.

During the visits, several simple assessments are done: a muscle‑function questionnaire called NSAD, a walking speed test over ten meters known as the 10MWT, a breathing test that measures how much air can be exhaled called FVC, an upper‑limb function test named PUL 2.0, a walking distance test over one hundred meters called 100MTT, and a blood test that measures a protein called CK which indicates muscle damage. These checks help researchers understand how the medication affects muscle strength, movement, and overall health over time.

The aim of the trial is to determine whether the combination of sarilumab and a 52‑week prednisone taper works better than prednisone taper alone in achieving lasting symptom control. Participants are randomly assigned to receive one of two dose levels of the study drug (150 mg or 200 mg) every two weeks, while all continue the steroid taper for one year, and they are followed for the same period to see how they respond.

Throughout the year, researchers check whether participants reach “remission,” meaning they have no significant pain or stiffness, and they record any side effects or laboratory changes. They also track how long remission lasts, whether symptoms return (a “flare”), and how the condition affects physical and mental well‑being using standard questionnaires. Safety is monitored continuously, and any serious problems are reported promptly.

Participants will be randomly assigned to receive either the active medication or the placebo for about one year, with regular clinic visits for injections and health checks. Blood samples will be taken to monitor liver enzymes (ALT and AST), a blood test called the ELF score that estimates liver scarring, and other measures such as cholesterol, blood sugar control (HbA1c), and body weight. Imaging tests, including a special ultrasound that measures liver stiffness (VCTE‑LSM), an MRI‑based scan (MRE), and an MRI that quantifies liver fat (MRI‑PDFF), will be performed at the start and at the end of the study to see how the liver is responding. Any side effects or abnormal lab results will be recorded throughout the study period.

The purpose of the trial is to determine whether MK-1045 can achieve a higher rate of complete remission (no detectable cancer) and improve overall survival (how long participants live) compared with blinatumomab. Participants will receive a series of treatment cycles lasting several weeks, during which doctors will monitor for adverse events (side effects) and check for minimal residual disease negativity (absence of tiny amounts of cancer cells). Some participants may later undergo allogeneic hematopoietic stem cell transplantation, a procedure that replaces diseased blood‑forming cells with healthy ones from a donor, if deemed appropriate by their physicians.

Participants will receive the assigned tablet daily and the infusion every few weeks, with regular clinic visits for safety checks, blood tests and imaging scans such as CT scans to look for changes in tumor size. The study will continue until the disease gets worse, a participant stops treatment, or the trial ends. Researchers will record how long the cancer stays stable (progression‑free survival), overall survival, side effects, and quality‑of‑life questionnaires.

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.

The purpose of the study is to compare the effects of Odevixibat with a placebo in children with Biliary Atresia. Participants will receive either Odevixibat or a placebo once daily. The study will last for up to 104 weeks, during which time the health of the participants will be closely monitored. The main focus will be on the time it takes for participants to require a liver transplant or experience other serious health issues related to their liver condition.

Throughout the study, various health parameters will be assessed, including liver function tests and overall health status. The study aims to provide valuable information on whether Odevixibat can help improve the outcomes for children with Biliary Atresia who have had the Kasai procedure. Participants will be regularly checked for any side effects or changes in their health to ensure their safety during the trial.