Muroran

Researchers are evaluating the safety and clinical effects of Awiqli Insulin Icodec in people with diabetes mellitus in Japan through a non-interventional observational study. The study focuses on real-world use of Awiqli prescribed by doctors as part of routine clinical care, with no restrictions on diabetes type or prior treatments. This research aims to gather information about adverse reactions and clinical outcomes over a one-year period. Participants will receive commercially available Awiqli once weekly, with treatment decisions made by their doctors following normal clinical practice. The study does not assign treatment but observes patients using Awiqli as prescribed. Data will be collected throughout the 52-week period to monitor safety and clinical parameters under real-world conditions. During the study, participants will be monitored for adverse reactions, serious adverse events, and changes in blood sugar control, including glycosylated hemoglobin HbA1c and fasting plasma glucose. Quality of life related to diabetes therapy will also be assessed. The study involves regular data collection over approximately one year, and participants continue with their usual care while contributing information to the study.

Spinal muscular atrophy SMA is a genetic disorder caused by changes in the SMN1 gene, leading to low SMN protein levels and problems with motor neurons. It is an inherited disease that mainly affects children and is a leading cause of infant death due to genetic conditions. This research aims to observe patients with SMA over time to understand long-term outcomes with new treatments and to evaluate the long-term safety and effects of the investigational therapy OAV-101. This is a prospective, multinational, non-interventional observational study where patients receive their usual clinical care without changes directed by the study. Treatments such as Zolgensma may be given according to normal clinical practice, but this is not controlled by the study protocol. Patients will be followed for up to 15 years or until death, with no extra visits or tests beyond what is normally done for SMA care. Participants will be evaluated through regular clinical assessments based on their normal care schedule, with data collected at baseline and at intervals of every 6 months for the first 2 years, then yearly for up to 15 years. Researchers will measure survival rates, motor function scores like CHOP-INTEND and HFMSE, hospitalizations, quality of life, use of ventilator, nutritional, and mobility supports, as well as any treatment-related side effects during this long-term follow-up.