Rare diseases
Choose from a range of resources covering rare and orphan diseases, from acute hepatic porphyria to late-onset Pompe disease. This hub includes interactive medical education tools, expert opinion, guideline recommendations, and the latest treatment options.
Gain expert insights into diagnosing and treating thymidine kinase 2 deficiency in pediatric patients – and earn 1 CME credit.
Latest resources
Myasthenia Gravis Learning Zone
Medical education resources focused on identifying early symptoms of myasthenia gravis, available treatments and information about the latest findings in new therapy pathways
Individualised treatment for MG
Read about Nils Erik Gilhus’ Moritz Romberg Lecture, real-world data on eculizumab and efgartigimod, and how gender impacts myasthenia gravis.
Improving outcomes in AL amyloidosis
Stay ahead in AL amyloidosis management. Access our congress updates and expert-led medical education content for the latest best practices.
Accredited symposium: Elevating LOPD care
Join Prof. Schoser, Dr. Mozaffar and Dr. Kishnani for an accredited symposium focused on translating patient perspectives into effective care.
Pompe Disease: Key findings from WMS 2022
Review developments in Pompe disease, expert opinion and discussion, and more in the following pages. Highlights and forthcoming expert content focus on the 2022 World Muscle Society Congress which was held in Halifax, Canada 11–15 October.