Accelerating therapies for rare neuromuscular disorders through European collaborations

The DREAMS consortium brings together leading institutions from across the continent to develop data-driven, patient-derived models for therapeutic discovery in rare NMDs.

UCL’s addition as the sole UK partner highlights the university’s longstanding commitment to collaborative European research. 

Neuromuscular diseases - which vary in severity and include conditions with life-threatening weakness impacting breathing muscles - affect approximately 150,000 people in the UK and around 14,000,000 people globally. 

Rare neuromuscular disorders - such as muscular dystrophies - remain one of the most underserved areas of medicine, with few approved therapies and limited tools for early diagnosis or intervention.  

Backed by €8 million Horizon Europe funding, including the UKRI Horizon Europe guarantee scheme, DREAMS aims to tackle these challenges by combining cutting‑edge lab techniques with stem‑cell research and AI tools that can highlight which potential medicines are most likely to work. 

In joining the consortium, the two UCL groups led by Professor Francesco Saverio Tedesco (UCL Biosciences, Great Ormond Street Hospital for Children and The Francis Crick Institute) and Dr Sara Benedetti (UCL GOS Institute of Child Health, Zayed Centre for Research into Rare Disease in Children) will contribute internationally recognised strengths in neuromuscular disease biology, stem cell engineering and translational medicine, building on links with academic, clinical, patient-led and industry partners. 

Professor Tedesco said: “We are delighted to join the DREAMS consortium to advance research into rare neuromuscular disorders. This collaboration allows us to combine UCL’s expertise in stem cell modelling, disease phenotyping and advanced therapies with the consortium’s collective strengths.” 

Dr Benedetti added: “By harnessing our strategic partnerships with the Francis Crick Institute and the NIHR Great Ormond Street Hospital Biomedical Research Centre, we aim to accelerate the translation of cutting-edge science into meaningful therapies for patients.” 

The UCL teams will contribute to DREAMS' work across three key areas: helping to spot early biological signs of rare neuromuscular diseases; creating new patient‑derived cell models that allow researchers to study these conditions in the lab; and testing potential new medicines - identified using AI - to see how well they work before they move on to more advanced stages of development. 

The consortium brings together a set of highly complementary organisations from across France, Belgium, Portugal, Israel and Spain. 

UCL’s entry into DREAMS also builds on its leadership of another major Horizon Europe-funded programme tackling neuromuscular disease: the MAGIC (Next-generation Models And Genetic Therapies for rare neuromuscular diseases) consortium. 

Led by Professor Tedesco and his research team at UCL and the Francis Crick Institute, with 16 other partners across ten countries worldwide, MAGIC received €10 million from Horizon Europe, UKRI and the Swiss SERI to develop advanced “muscle-in-a-dish” models for testing next-generation gene therapies.  

MAGIC aims to create more predictive human cell-based models of muscular dystrophy to improve the safety and effectiveness of gene therapy approaches, reduce reliance on animal models, and accelerate new treatments for conditions such as Duchenne muscular dystrophy (DMD), X-linked myotubular myopathy and other severe congenital muscular dystrophies. 

Among key partners are Inserm - the only public research organisation in France entirely dedicated to human health - which is involved in both consortia, with Dr. Mario Amendola (Inserm and Genethon) and Dr Xavier Nissan (iStem and Inserm) supporting coordination of both MAGIC and DREAMS. Inserm is UCL’s largest partner in France by co-publications and in 2025 the two institutions signed a new Joint Lab agreement - Inserm's first with a UK organisation. 

Links 

• UCL joins the DREAMS consortium: Strengthening Europe’s scientific capacity to accelerate therapies for rare neuromuscular diseases 
• ‘Muscles in a dish’ project to test genetic therapies for muscular dystrophy 
• UCL Division of Biosciences 
• UCL Cell and Developmental Biology 
• UCL Faculty of Life Sciences 
• UCL Population Health Sciences 
• UCL GOS Institute of Child Health 
• Zayed Centre for Research into Rare Disease in Children 
• The Francis Crick Institute 
• DREAMS website 
• MAGIC website 
• Inserm
• The Tedesco laboratory

Images

UCL's Professor Francesco Saverio Tedesco and Dr Sara Benedetti; One of the stem cell-derived 'mini muscles' developed as part of the MAGIC consortium. Credit: Terry Pengilley/MDUK.

Media contact

Sophie Vinter

Tel: +44 (0)20 3108 7787

Email: s.vinter [at] ucl.ac.uk