Myasthenia Gravis: the PANORAMA Project wins €500,000 in funding to study refractory forms

Refractory myasthenia gravis, a rare and complex autoimmune disease for which a significant proportion of patients do not respond to available therapies, is the focus of the PANORAMA project—developed at the Policlinico of Milan—funded through the “Collaborative Research Under 40” call promoted by the Regional Foundation for Biomedical Research (FRRB).

Myasthenia gravis is an autoimmune disease that causes fluctuating muscle weakness, driven by antibodies that attack the acetylcholine receptor (AChR), impairing neuromuscular transmission. Approximately 10–15% of patients develop a refractory form that does not respond to conventional treatments, with frequent exacerbations, hospitalizations, and intensive care admissions, resulting in a significant impact on quality of life and high healthcare costs. However, the mechanisms underlying treatment resistance are still not clearly understood, and early predictive biomarkers are currently unavailable in clinical practice.

PANORAMA (From PAtients to Neuromuscular Organoids in Refractory AChR+ Myasthenia grAvis) aims to fill this gap. The project is coordinated by Delia Gagliardi (neurologist and Principal Investigator) and Federica Rizzo (researcher and Co-Principal Investigator) at the Policlinico of Milan, in collaboration with Yuri Matteo Falzone, neurologist at IRCCS San Raffaele Hospital. The study has been funded by the “Collaborative Research Under 40” call of the Regional Foundation for Biomedical Research (FRRB) with a grant of €500,000. The initiative was created to promote collaboration among researchers under 40 and supports translational research projects with potential impact on the regional healthcare system.

The research will involve 30 patients and 10 healthy controls without myasthenia gravis to develop neuromuscular organoids—patient-specific cellular models capable of recreating the neuromuscular junction in vitro. These organoids, derived from induced pluripotent stem cells, will be used to study endplate damage and identify new molecular targets through advanced morphological, functional, and multi-omics analyses. The goal is to identify markers of refractoriness and new therapeutic targets, enabling earlier access to innovative treatments and advancing a personalized medicine approach.

This represents an important recognition of the work carried out by researchers at the “Centro Dino Ferrari” of the University of Milan – Policlinico of Milan, led by Professor Giacomo Comi, Director of Neurology at the hospital and faculty member at the University of Milan, in close synergy with the Neurology Unit of IRCCS San Raffaele Hospital, directed by Professor Massimo Filippi, full professor at Vita-Salute San Raffaele University.

The collaboration between the two IRCCS institutes confirms their leading role in translational research on neuromuscular diseases, strengthening the bridge between laboratory research and patient care.