Thirty clinicians, physiotherapists and nurses from multidisciplinary teams across the UK came together to share experiences and to discuss the continued implementation of a consistent monitoring approach for patients with late-onset Pompe disease (LOPD).
The workshop focused on the use of patient-reported outcome measures (PROMs) and ventilator data for monitoring, and implementation of the North Star Assessment for limb-girdle type muscular dystrophies (NSAD) in routine clinical practice. Patients and patient organisations discussed their experience and needs for disease monitoring, and representatives from metabolic centres shared their learnings and best practice.
The report is available here: P POM UKI PDMW 2026 Meeting Report June 2026