Ciliopathy

This early-stage trial tests a single injection of AXV-101 gene therapy into one eye of 12 children and teens (ages 4-17) with Bardet-Biedl syndrome type 1, a rare genetic condition that causes progressive vision loss. The main goal is to check safety and find the right dose, whi…

This study tests whether children aged 6–12 with chronic lung infections (like bronchiectasis) can improve their exercise ability and lung clearance using online physiotherapy sessions. Over 12 weeks, 32 children will either receive remote guided breathing exercises and workouts …

This study tests a new CPR method called Bio-CPR, which synchronizes chest compressions with ventilation using a machine. Researchers will compare it to standard CPR in 408 adults who have a witnessed cardiac arrest. The goal is to see if this coordinated approach improves breath…

This study looks at whether adding a continuous saltwater mist to warm, humidified air can make thick lung mucus easier to clear. 35 adults hospitalized with lung conditions like COPD or cystic fibrosis will receive both treatments on separate days. Researchers will measure mucus…

This study works with patients and healthcare professionals to create educational resources about fertility and pregnancy for people with Primary Ciliary Dyskinesia (PCD). The goal is to develop a patient-friendly guide and a toolkit for doctors. The study will involve 30 partici…