currently in the phase 1/2 FORTIS study in late-onset Pompe disease (LOPD) in adults. Xork is designed as a pre-treatment to overcome one of the key limitations to adeno-associated virus (AAV ...

How common are genetic diseases in children, young people and adults treated for Myalgic Encephalomyelitis / Chronic Fatigue Syndrome (ME/CFS)?‌ We are looking for individuals with a diagnosis of ...