This study investigates the effectiveness of different investigational medications compared to a placebo in improving lung capacity in participants with Interstitial Lung Disease (ILD) secondary to Systemic Sclerosis (SSc). The purpose is to observe changes from the start to the end of the treatment period at Week 52.Participants in the …
This study investigates the safety and effects of an investigational mRNA vaccine for individuals with mild acne. The study will include two groups, a Sentinel Cohort to initially assess safety, and a Main Cohort for broader evaluation.Participants will receive two administrations of the investigational vaccine. The study will consist of …
This study investigates the effects of a one-time injection for people with Geographic Atrophy (GA) due to Age-related Macular Degeneration (AMD).The purpose of this study is to evaluate the safety and effectiveness of the injection in treating GA. Researchers will observe how the treatment impacts the condition over time.Who can …
This study investigates the safety and immune response of a 21-valent pneumococcal conjugate vaccine in children and adolescents with sickle cell disease. The goal is to see if the vaccine can help the body make antibodies, which are special proteins that fight germs.The study focuses on comparing the investigational vaccine …
This study investigates the experiences of adults with relapsed or refractory multiple myeloma. The purpose is to gather data about patients receiving standard care with an investigational medication. Researchers aim to collect various types of data, including quantitative, qualitative, and wearable data, to understand the treatment experience better.Participants will be …
This study investigates the administration of an investigational medication in people with multiple sclerosis (MS). The purpose is to compare two methods of giving the medication: under the skin (subcutaneous) and into a vein (intravenous).The study focuses on evaluating the effectiveness and safety of these two administration methods. Participants will …
This study investigates Gaucher disease, aiming to understand its variability and progression to improve therapeutic interventions.The study also focuses on evaluating pregnancy outcomes in women with Gaucher disease. It aims to track complications and infant growth to enhance patient care.Who can participate: Adults and children with confirmed Gaucher disease can …
This study investigates Pompe disease, focusing on how it progresses over time in individuals diagnosed with the condition.The study aims to understand the variability and progression of Pompe disease. It seeks to gather information to aid in developing patient care recommendations and evaluating the effectiveness of long-term treatments.Who can participate: …
This study investigates Fabry disease, focusing on observing and collecting data on individuals diagnosed with the condition, regardless of their treatment status, to better understand the disease's progression and impact.The study aims to gather information on the safety and effectiveness of treatments for Fabry disease, as well as to track …
This study investigates the pregnancy outcomes in women with Pompe disease. The purpose of this study is to track outcomes during pregnancy, including complications and infant growth, for women with this condition.The study focuses on understanding how pregnancy progresses in women with Pompe disease, whether they receive disease-specific treatments or …
