This study investigates the effects of an investigational medication on individuals with infantile-onset Pompe disease (IOPD). Pompe disease is a rare genetic disorder caused by the buildup of a complex sugar called glycogen in the body's cells. This buildup impairs the function of various organs and tissues, especially muscles. The …
This study investigates the effectiveness and safety of an investigational medication in adults with chronic pruritus of unknown origin (CPUO). Pruritus is a medical term for severe itching. The study involves two separate treatment arms, where participants will receive either the investigational medication or a placebo. A placebo is an …
This study investigates the effect of an investigational medication on heart health in adults with Fabry disease, a genetic disorder that affects the body's ability to break down a specific type of fat. This can lead to a buildup of fat in the body's cells, causing various symptoms, including heart …
This study investigates the use of an investigational medication for treating adults with moderate-to-severe asthma. Asthma is a condition where the airways in the lungs become inflamed and narrow, making it difficult to breathe. The study aims to understand the long-term safety, tolerability, and effectiveness of this medication in participants …
This study investigates the safety and effectiveness of an investigational medication in people with moderate to severe atopic dermatitis, a skin condition that causes itchy and inflamed skin. Participants in this study have previously been involved in a clinical trial for this investigational medication and will continue to be monitored …
This study investigates the effect of an investigational medication compared to standard care in preventing or slowing lung function decline in adults with uncontrolled moderate to severe asthma. Asthma is a condition where the airways in the lungs become inflamed and narrow, making it difficult to breathe. The study aims …
This study investigates the effects of an investigational medication given as an add-on therapy for adults with moderate-to-severe asthma. Asthma is a condition where the airways in the lungs become inflamed and narrow, making it hard to breathe. The study aims to assess how well the medication works, its safety, …
This study investigates the safety and effectiveness of an investigational medication for people with newly diagnosed Type 1 Diabetes (T1D) who are already using insulin. Type 1 Diabetes is a condition where the body does not produce insulin, a hormone needed to allow sugar to enter cells to produce energy. …
This study investigates the safety and tolerability of an investigational medication for individuals with Chronic Obstructive Pulmonary Disease (COPD). COPD is a long-term lung disease that makes it hard to breathe. The study involves participants who have completed previous clinical studies related to this condition. Participants will receive the investigational …
This study investigates the effects of an investigational medication on pain symptoms in individuals with Fabry disease. Fabry disease is a genetic condition that can cause pain in the nerves and abdomen. Participants in this study must be 16 years or older and have Fabry disease but have not received …