This study investigates the safety and efficacy of an investigational gene therapy for individuals with non-congenital myotonic dystrophy type 1 (DM1). The purpose of this study is to assess how well the investigational therapy can reduce levels of a specific protein linked to DM1 and improve muscle function in participants …
This study investigates the treatment of moderate-to-severe atopic dermatitis in participants aged 12 years and older. The purpose is to evaluate the efficacy and safety of an investigational medication given as a subcutaneous injection, compared to a placebo. Participants will continue using their topical corticosteroids (TCS) during the study. The …
This study investigates the safety and effectiveness of an investigational medication compared to a placebo in adults with chronic obstructive pulmonary disease (COPD) characterized by an eosinophilic phenotype. Participants will be involved in the study for up to 60 weeks, which includes different phases like screening, treatment, and follow-up. During …
The purpose of this study is to investigate the effects of investigational medications on primary focal segmental glomerulosclerosis (FSGS) and primary minimal change disease (MCD) in participants aged 16 to 75 years. The study will last up to 76 weeks, and participants will be involved for this entire duration. FSGS …
This study investigates the safety, efficacy, and immune response of an investigational mRNA vaccine for adults with moderate to severe acne. This study aims to evaluate how well the vaccine works at different doses in participants aged 18 to 45 years.Participants in the study will receive up to three intramuscular …
This study investigates an investigational medication for metastatic castration resistant prostate cancer (mCRPC). The purpose of this study is to understand the safety, how the body processes the investigational medication, and its preliminary effectiveness in treating mCRPC.Participants in the study will receive the investigational medication in different parts. Initially, there …
This study investigates the clinical outcomes of patients with Gaucher disease. The study is observational, meaning it collects data from patients receiving routine care without altering their treatment. It aims to enhance understanding of the disease's variability, progression, and natural history to aid in better therapeutic interventions.Participants will undergo clinical …
This study investigates Pompe disease, focusing on muscle function impacts due to glycogen buildup. The purpose of this observational study is to track the natural history and outcomes of Pompe disease in patients worldwide, regardless of treatment status. An observational study involves collecting data by observing participants receiving routine care, …
This study investigates Fabry disease, focusing on tracking the routine clinical outcomes for patients, regardless of their treatment status. The study is observational, meaning it collects data by observing participants without altering their treatment. Participants will be in the study for an ongoing period as determined by their physician.Participants in …
