In this new edition of “Meet the Team”, we travel to Paris to meet one of Europe’s leading medical institutions: Assistance Publique–Hôpitaux de Paris (AP-HP). As a cornerstone of the DREAMS consortium, AP-HP brings together exceptional clinical experience, patient care, and biomedical innovation to accelerate progress in rare neuromuscular disease research.
AP-HP: A world-class institution at the crossroads of care, education, and science
AP-HP is the largest Academic Medical Center in Europe, affiliated with six universities and comprising 38 hospitals organised into six main hospital systems. Representing 10% of France’s public hospital sector, it provides state-of-the-art care to more than 8 million patients each year.
Its mission rests on three inseparable pillars: delivering the highest quality of care, training the next generation of healthcare professionals, and advancing biomedical research at the highest international standards. AP-HP also leads in clinical innovation, producing more than half of all clinical research in France and registering numerous patents every year.
Driving DREAMS Work Packages 1 and 6
Within DREAMS, AP-HP plays a pivotal role in two major Work Packages:
- WP1 – Biological Materials in 5 NMDs for Screening, Proteomic Data, and New Biomarkers
The AP-HP team leads the collection and analysis of biological samples to identify shared biomarkers across five neuromuscular diseases (NMDs). This work builds on collaboration with external partners, including the European Reference Network for Neuromuscular Diseases (ERN Euro-NMD) coordinated by Professor Evangelista.
Their goal is to bridge basic research and patient data — characterising skeletal muscle biopsies to translate molecular findings into clinical understanding.
- WP6 – Towards Therapy: Design an Adaptive Clinical Trial
Together with other partners, AP-HP is shaping innovative clinical trial designs tailored to the realities of rare diseases. With small and geographically dispersed patient populations, traditional trials often fall short. The team is pioneering basket trial models that test a single therapy across multiple diseases sharing common biological mechanisms, improving efficiency and statistical robustness.
These efforts directly support DREAMS’ overarching mission: to create a reusable, AI- and iPSC-powered framework for faster, safer, and more effective treatments for rare neuromuscular disorders.
Meet the AP-HP experts behind the impact
Professor Evangelista – From genetic mechanisms to patient-centred research
Professor Evangelista is a Professor of Neuromuscular Diseases at AP-HP, specialising in the genetics, pathophysiology, and therapy of NMDs. Her work connects molecular biology with patient care — a hallmark of DREAMS’ approach.
She has published extensively on the genetic mechanisms underlying disorders such as Duchenne Muscular Dystrophy (DMD) and Facioscapulohumeral Muscular Dystrophy (FSHD), helping to elucidate how mutations drive disease progression. Her research also explores gene therapy and pharmacological interventions, often using induced pluripotent stem cells (iPSCs) to model diseases and test promising compounds.
Beyond the lab, Professor Evangelista is a strong advocate for patient engagement. She collaborates with organisations and networks, including ERN Euro-NMD, to ensure patients’ voices are central in research and clinical decisions.
Professor Karim Wahbi – Advancing cardio-neurology in rare diseases
A globally recognised expert in cardio-neurology, Professor Karim Wahbi serves as a Senior Cardiologist at the Reference Centre for Neuromuscular Diseases (Hôpital Cochin and Hôpital Européen Georges-Pompidou, AP-HP)**. His pioneering work has transformed the management of cardiac complications in neuromuscular disorders.
As a Principal Investigator at the Institut Cochin (INSERM U1016), Professor Wahbi bridges clinical practice and molecular research, focusing on the mechanisms and management of cardiac involvement in muscular diseases such as Myotonic Dystrophy Type 1 (DM1). His landmark studies have reshaped international guidelines, improving survival and quality of life for patients.
He also contributes to the BIND Phase 3 clinical trial evaluating Laquinimod for DM1, ensuring cardiac outcomes remain at the forefront of therapeutic research. His leadership extends to mentoring young clinicians and fostering interdisciplinary collaboration — values deeply aligned with DREAMS’ mission.
AP-HP: Collaborating for the future of rare disease research
Through its dual role in clinical excellence and research innovation, AP-HP exemplifies how multidisciplinary teamwork can bridge the gap between discovery and therapy. By contributing to shared biomarker identification, adaptive trial design, and patient engagement, AP-HP strengthens DREAMS’ ambition to make precision medicine a reality for patients with rare neuromuscular diseases.
“AP-HP’s strength lies in its ability to connect research, clinical practice, and patient care within a single integrated ecosystem.”
