Latest Articles

What challenges do clinicians face in monitoring in disease progrission and response to therapy?

What factors influence your decision to switch a patient from alglucosidase alfa to avalglucosidase alfa?

Have you observed any significant differences in treatment outcomes between these two therapies in your local practice?

How has avalglucosidase alfa impacted the treatment paradigm for both infantile-onset and late-onset Pompe disease in your experience?

Pompe Care Podcast Series 2

Welcome to the Pompe Care podcast series. Today we speak with Dr. Rawda Sunbul, Consultant Medical Genetics at Qatif Central Hospital in Saudi Arabia, about Pompe disease. We discuss the typical patient journey and diagnostic challenges. Dr. Sunbul shares insights on new diagnostic technologies, effective ways to support patients and families, and how to address the social stigma surrounding rare diseases.

Pompe Care Podcast Series 3

Today we speak with Dr. Nadia Alhashmi, Senior Consultant in Chemical & Bio-Chemical Genetics at the National Genetics Royal Hospital in Oman, and Dr. Khalid Althihli, Senior Consultant in Chemical & Bio-Chemical Genetics at Sultan Qaboos University Hospital in Oman. With deep expertise in rare metabolic and genetic disorders, both specialists bring frontline clinical perspectives on Pompe disease — covering the typical patient journey, diagnostic challenges, and new diagnostic technologies, while sharing their compassionate approach to supporting patients and families and addressing the social stigma surrounding rare diseases.

Structured Monitoring for Early Detection of Fabry Progression

Renal Outcomes After Switching to Agalsidase Beta: A Single-Center Analysis

Dupilumab for Patients with AFRS