POMPE Survey

A biotech company that conducts research and develops treatments for people living with rare diseases such as Pompe disease wants your help. They are recruiting Pompe patients and caregivers to take a short online survey about your knowledge and understanding of...

CLINICAL TRIALS/STUDIES

  STRIDE STUDY                            A Study in Subjects With LOPD Who Are Currently Being Treated With ERT   VAL-1221 for Pompe Disease  VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Patients With Late-Onset Pompe Disease neoGAA        ...