The purpose of the trial is to find out whether taking colchicine each day can slow the typical decline in muscle function compared with the placebo.

Participants will be randomly assigned to receive either the colchicine tablet or the placebo tablet for about 30 weeks, followed by an optional open‑label period where all may receive the active drug. During the study, they will attend regular clinic visits where basic health checks, breathing tests, and questionnaires about daily abilities and quality of life are completed. The study ends after a total of roughly 66 weeks, at which point the collected information will be used to evaluate the medication’s effect.

The purpose of the study is to show that switching to oral therapy after seven days is not worse than switching after fourteen days. Participants will receive IV antibiotics for either seven or fourteen days, then continue with oral medication for the remaining treatment period. After the antibiotics are stopped, health will be checked for up to 24 weeks to see if the infection returns, if additional spine surgery is needed, or if any serious problems occur. Quality of life and ability to perform daily activities will also be recorded during the follow‑up.

Participants will be followed for up to 24 months. Some will slowly reduce their medication doses (the tapering group), while others will continue taking their medicines as they have been (the usual‑care group). Throughout the study, regular visits will include simple checks of joint pain and swelling, blood tests, and a score called DAS28-CRP, which combines the number of tender and swollen joints with a blood marker of inflammation to show how active the arthritis is. Safety will be monitored, and the study will compare how much medication each group saves and whether disease activity stays similar over time.

The purpose is to determine whether the weekly treatment works at least as well as the daily treatment in promoting growth.

Participants will be assigned to receive either the weekly Somatrogon or the daily Genotropin for about twelve months. Their height will be measured regularly, and the main outcome will be the change in growth speed over the year, referred to as annualized height velocity. No additional procedures beyond the injections and height checks are required.

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 purpose of the study is to see whether LTG-321 reduces knee pain better than the placebo. This phase‑2 trial is randomized, meaning participants are assigned to treatment order by chance, and double‑blind, so neither the participants nor the study staff know which tablet is being taken at any time. It uses a crossover design, so each participant receives both the active tablet and the placebo in two separate periods lasting about two weeks each, with a short break in between. Throughout the study, participants keep a daily pain diary and complete the WOMAC questionnaire, which asks about pain, stiffness, and ability to perform daily activities. Safety checks include regular measurements of vital signs, an ECG (a simple heart rhythm test), and basic laboratory tests.

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 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.

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.

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 are randomly assigned to receive either tablets containing 1800 mg of thiamine hydrochloride or matching placebo tablets for a period of four weeks. The trial is conducted in a double-blind manner, meaning that neither the participants nor the study staff know which tablets are being taken. Study visits include an initial assessment, regular check‑ins, and a final evaluation after the treatment period.

The purpose is to compare the effect of high dose thiamine with placebo on changes in fatigue as measured by the Bristol Rheumatoid Arthritis Fatigue Multi-Dimensional-Questionnaire global score from the start of the study to week 4.

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.

The purpose of the trial is to see whether giving tranexamic acid during surgery changes the rate of post‑operative infections within one year. After the procedure, participants are followed for up to twelve months with check‑ups at two weeks, three months and one year. During these visits doctors look at how the wound is healing, whether the skin has opened up (wound dehiscence), how much blood loss occurred by measuring blood‑type protein levels, and if any additional surgery is needed. Bone healing (fusion) is checked at the final visit using a special imaging test called computed tomography, which produces detailed pictures of the bones. All participants receive the same standard care apart from the study medication, and the study records any infections, wound problems, extra surgeries, and the overall success of the bone fusion.

The purpose of the trial is to determine which method provides better shoulder function after three months. Participants receive a single injection session and are then asked to return for check‑ups at one month, three months, six months, and twelve months, during which their shoulder movement, pain level, and ability to resume normal activities are recorded.

Participants will take the assigned drug pair for several months while continuing usual care. During the study they will have regular visits for blood and urine samples, simple pain questionnaires, and brief physical checks. The laboratory work will look at levels of inflammation‑related proteins and cytokines to understand any changes, and safety will be monitored by recording any unwanted effects or abnormal test results. The overall goal is to determine whether the drug combinations can reduce pain, lower inflammation, and affect the aging markers measured at the start and end of the trial.

The purpose of the study is to determine whether the drug improves pain levels after 12 weeks compared with placebo. Participants receive an infusion at the start of the study, then again at about 12 weeks and 24 weeks, with follow‑up visits extending to 36 weeks. At each visit, pain is recorded using simple age‑appropriate scales that involve faces or a line marked from “no pain” to “worst pain.” Whole‑body imaging with MRI (a scan that creates detailed pictures of the bones) is performed to see how many bone lesions are present. Safety checks include monitoring for flu‑like symptoms, headache, and low levels of phosphate or calcium in the blood.

Participants who join the trial receive the assigned infusion and then return for regular check‑ups over the next 18 months. During these visits, simple tests are performed to measure how quickly a person can stand up, walk a short distance, climb stairs, and breathe fully. A small wearable device may be used to record walking speed in everyday life. Blood and muscle samples may be taken to look for the presence of a protein called microdystrophin, which the therapy is meant to produce. Safety is also monitored by recording any side effects, checking heart rhythm with an ECG, and listening to the lungs with an echo.

The study includes people with Duchenne muscular dystrophy whose gene changes may allow a treatment approach called exon 44 skipping, which means the medicine is designed to help the body work around a missing or faulty part of the gene. The study is planned in two parts. In the first part, treatment is assigned by chance and neither the study team nor the participant knows which treatment is given. Later, there is an open-label extension, which means the treatment is given openly after the blinded part ends. During the study, the medicine is given at regular visits and health checks are done over time.

The study is a long-term extension, which means it follows people over a longer period after earlier treatment. During the trial, the medicine is taken by mouth and participants are watched over time for side effects and other health changes. Regular checkups and laboratory tests are used to follow general health, joint symptoms, and skin symptoms while treatment continues.

The main aim of the trial is to find out whether either medication can lower the risk of developing new kidney stones and slow the progression of the disease. Participants will be followed for up to two years, with regular clinic visits where blood and urine samples are taken and simple imaging scans are performed to look for stones and assess kidney health. One of the key laboratory measures is the eGFR, which estimates how well the kidneys are filtering waste from the blood.

During the study, researchers will compare the occurrence of kidney stones, changes in kidney function, and overall physical and mental well‑being between the groups receiving the study medicines and those receiving a placebo. The information gathered will help determine if these treatments are useful for preventing stone formation and preserving kidney health in people with this condition.

The purpose of the study is to demonstrate the efficacy of maintaining RTX as a maintenance strategy in individuals with stabilized SSc‑ILD following RTX induction treatment. Participants receive the assigned infusion every few months for a total period of about 18 months, with clinic visits scheduled at regular intervals to monitor health.

During the study, lung function is checked using measurements such as FVC, which assesses the amount of air a person can forcefully exhale, and DLCO, which evaluates how well oxygen passes from the lungs into the blood. Imaging of the chest is performed with a HRCT scan, and physical capacity is measured with a 6‑min walk test that records the distance walked in six minutes. Additional assessments include questionnaires about quality of life and safety checks.

People in the study are assigned by chance to receive either tulisokibart or placebo, and neither the participants nor the study team knows which treatment is given during the study. Treatment is given over a period of time, and the main comparison is made after about 16 weeks. During the study, health checks are done to see how the disease and any side effects change over time.

During the study, participants will receive either the active medication or a placebo. The treatment lasts for 48 weeks. While receiving the treatment, various observations will be made to monitor how the body responds and to check for any adverse events, which are unwanted or harmful medical occurrences. Monitoring includes checking vital signs, such as heart rate and blood pressure, and performing an electrocardiogram to check the electrical activity of the heart.

Participants will receive QEL-005 through an intravenous infusion, which is a method of delivering the medicine directly into a vein. The study is conducted in stages to monitor how the body reacts to the treatment. During this process, health professionals will monitor for any side effects or changes in vital signs and electrocardiogram, a test that records the electrical activity of the heart, to ensure the safety of the procedure.

The treatment involves applying clobetasol propionate directly to the skin of the hands. Participants in the study will follow a specific schedule where the medication is used twice a day for the first two weeks, followed by a period of using it once a day for the next two weeks. This approach is known as a tapering regimen, which means the amount or frequency of the medication is gradually reduced over time.

The purpose of the study is to determine whether the combination tablet provides better pain relief and is well tolerated compared with the single‑ingredient tablet and placebo. Over a period of about one week, participants take the assigned tablets at regular intervals and record their pain levels and any need for extra pain medication. Simple physical tests, such as reaching toward the floor, are performed at the start and at the end of the week to see if movement improves.

The purpose of the study is to determine whether the injection can reduce knee pain and improve function compared with the placebo. Participants will receive a single injection and then be asked to complete simple questionnaires about how their knee feels, how much it hurts, and how well they can perform daily activities. These questionnaires include the Western Ontario and McMaster Universities Arthritis Index (WOMAC), which scores pain, stiffness and functional ability on a scale from 0 to 100. Over the following months, researchers will also keep an eye on any adverse events or side effects and may take routine lab tests to check safety.

The study involves comparing the effects of bimekizumab (also referred to as BZK) and adalimumab (also referred to as ADA). Bimekizumab is a medication that works by blocking two specific proteins in the body that contribute to inflammation. The participants will receive one of these two treatments through an injection. During the course of the study, the way these medications manage pain and inflammation in the spine and chest area will be observed over several months.