Rare neuromuscular diseases affect relatively small patient populations, yet their impact on patients, families and healthcare systems across Europe is substantial. Many patients experience long diagnostic journeys and, for numerous rare neuromuscular conditions, therapeutic options remain limited, with few disease-modifying treatments available. Addressing these challenges requires not only scientific excellence, but also new collaborative approaches that bridge biology, data science and clinical research.
In this context, the EU-funded DREAMS project (Drug REpurposing with Artificial intelligence for Muscular disorderS) has been featured by CORDIS, the European Commission’s platform for showcasing research and innovation results. The article highlights DREAMS’ integrated research approach and marks an important milestone for the project with the official inclusion of University College London (UCL) in the consortium.
The scientific and clinical challenge of rare neuromuscular diseases
Rare neuromuscular diseases comprise a diverse group of disorders affecting muscle structure, function and maintenance. While their clinical presentation varies, patients often share common challenges: delayed diagnosis, progressive muscle degeneration and limited treatment options.
From a research perspective, rarity poses significant obstacles. Small patient cohorts restrict data availability, while traditional drug development pipelines are often too lengthy and costly to be viable for rare diseases. As a result, promising scientific discoveries frequently struggle to translate into therapies that reach patients.
DREAMS was conceived to respond to these challenges by rethinking how therapies for rare neuromuscular diseases can be identified, prioritised and evaluated, combining biological relevance with computational scale.
DREAMS: combining AI, stem cells and phenotypic screening
Funded under Horizon Europe and launched on 1 November 2023, DREAMS brings together expertise in artificial intelligence, stem cell biology and neuromuscular disease research to accelerate early-stage therapeutic discovery.
At the core of the project is the integration of AI-based drug discovery tools with induced pluripotent stem cell (iPSC) models derived from patients. These models allow researchers to study disease mechanisms in human muscle cells and to perform advanced phenotypic screening in a controlled and scalable way.
DREAMS focuses initially on five rare neuromuscular diseases:
- Centronuclear myopathy
- Duchenne muscular dystrophy
- Emery-Dreifuss muscular dystrophy type 2
- Pompe disease
- Danon disease
These conditions were selected because they share underlying pathophysiological features, including disruptions linked to autophagy-related cellular processes. By studying them together, DREAMS aims to identify shared biomarkers and molecular pathways that could inform therapeutic strategies across multiple diseases.
Rather than approaching each disorder in isolation, the project explores whether common mechanisms can be targeted, creating efficiencies that are particularly important in the rare disease context.
Strengthening the consortium: the contribution of University College London
As highlighted in the CORDIS article, the DREAMS consortium has been strengthened by the addition of University College London (UCL), a leading European institution in biomedical research and regenerative medicine.
Within DREAMS, UCL contributes high-quality patient-derived iPSC lines for additional neuromuscular diseases, enhancing the diversity and robustness of the project’s experimental models. The team also brings advanced expertise in muscle differentiation and cellular modelling, which is essential for analysing disease mechanisms and validating biomarker signatures.
In addition, UCL contributes specialised assays to assess drug efficacy and mechanisms of action, alongside deep clinical and scientific knowledge of neuromuscular disorders. These capabilities reinforce DREAMS’ capacity to connect computational predictions with experimentally and clinically meaningful insights.
As noted by UCL researchers Francesco Saverio Tedesco and Sara Benedetti, the collaboration allows the consortium to combine complementary strengths and leverage strategic partnerships with organisations such as the Francis Crick Institute, the NIHR Great Ormond Street Hospital Biomedical Research Centre and the MAGIC consortium.
European collaboration driving innovation in rare disease research
DREAMS exemplifies the value of European research collaboration in addressing complex health challenges. Supported by Horizon Europe, the project brings together academic institutions, research hospitals, technology providers and patient-focused organisations from across Europe.
This interdisciplinary structure ensures that AI tools are developed in close dialogue with biomedical researchers and clinicians, while remaining attentive to ethical, regulatory and societal considerations. The recognition by CORDIS reflects the European Commission’s commitment to promoting research initiatives that combine scientific excellence with clear societal relevance.
Sharing knowledge and looking ahead
DREAMS researchers have begun sharing the project’s approach and early insights with the wider scientific community. In November 2025, the consortium presented its work at the 22èmes Journées de la Société Française de Myologie in Aix-les-Bains, France, contributing a talk on shared molecular mechanisms across myopathies and a poster outlining the project’s structure, objectives and methodology.
While DREAMS is still ongoing, its ambition is to build a robust scientific and methodological foundation that supports future therapeutic development. Rather than promising immediate clinical outcomes, the project focuses on generating knowledge, tools and collaborative frameworks that can shorten and strengthen the path from discovery to patient benefit.
A milestone for DREAMS
Being featured by CORDIS marks an important moment for DREAMS, recognising both the project’s innovative approach and the growing strength of its consortium. More broadly, it underscores the role of coordinated European research in advancing new ways to study and address rare neuromuscular diseases.
As DREAMS continues, its work aims to contribute to a future where research into rare neuromuscular conditions is faster, more connected and more responsive to patient needs — grounded in science, guided by data and enabled by European collaboration.
