- Muscular Dystrophy UK (MDUK) supports high quality research that drives rapid access to emerging therapies for people with muscle-wasting conditions.
- Facioscapulohumeral muscular dystrophy (FSHD) is a rare muscle-wasting disease affecting an estimated 3-5 in every 100,000 people worldwide.
- This partnership will combine Healx’s AI-driven drug discovery expertise with MDUK’s patient insight to identify novel therapeutic options for FSHD.

Cambridge UK – 15 July 2020 – Healx, the AI-powered, patient-inspired technology company accelerating the discovery and development of rare disease treatments at scale, today announced its Rare Treatment Accelerator Programme (RTA) partnership with Muscular Dystrophy UK (MDUK). The RTA collaboration will initially focus on finding novel therapies for facioscapulohumeral muscular dystrophy (FSHD) - a genetic condition that causes muscle-weakening and wasting in the face, shoulder blades and upper arms - with an aim to explore other muscular dystrophies in future projects.

With an estimated 3-5 people in every 100,000 affected globally, FSHD is one of the more common forms of muscular dystrophy. In the United Kingdom alone, there are between 2,000-2,500 people living with the condition, but there is no cure and a lack of effective treatments means new therapeutic options are urgently needed. By combining MDUK’s extensive patient insights and Healx’s AI-powered drug discovery expertise, the partnership aims to develop new therapies for the condition.

Healx’s innovative approach integrates the power of big data and artificial intelligence with expertise from the in-house expert pharmacology team to uncover new connections between existing drugs and diseases. This approach reduces the time, cost and risk of traditional drug discovery methods – increasing the potential for accelerated access to more cost-effective treatments for patients.

Commenting on the partnership, Dr Kate Adcock, Director of Research and Innovation at Muscular Dystrophy UK, said: “We’re really excited about this RTA partnership, and look forward to working with Healx to identify potentially promising therapies for FSHD. It’s an exciting time for research into muscle-wasting conditions, and treatments are on the horizon. By working with world-leading partners like Healx, we can drive vital research forward and accelerate access to treatments.”

Dr Bruce Bloom, Chief Collaboration Officer at Healx, says: “Healx is proud to be working with MDUK, the UK’s leading charity for muscular dystrophies, to combat the lack of currently available treatments for FSHD. Having successfully carried out drug matching projects for similar conditions, the team is looking forward to applying their expertise in neurological and neuromuscular diseases to find a treatment for this life-altering condition. We are looking forward to sharing more updates about this project in the coming months. ”

This Rare Treatment Accelerator Programme partnership is the latest in a series of RTA collaborations between Healx and patient groups. In April, the company announced that it would be working with the Children’s Tumor Foundation to develop new therapies for neurofibromatosis - a rare genetic disorder that affects 1 in 3,000 people worldwide. The team is also working with US-based patient group FRAXA Research Foundation to find new treatments for Fragile X syndrome, the most common inherited cause of autism and intellectual disabilities in the world. Working in collaboration, they have progressed a number of new therapies from the drug prediction stage to being ready for clinical testing - in just 24 months.

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About Healx
Healx is an AI-powered, patient-inspired technology company, accelerating the discovery and development of rare disease treatments at scale. There are 7,000 known rare diseases that affect 350 million people across the globe but only 5% of those conditions have approved treatments. Healx’s mission is to identify and progress novel therapies for the 95% of rare diseases without approved treatment.

To do this, Healx combines AI technology with drug discovery expertise and Rare Treatment Accelerator Programme patient insight to identify existing drugs that may be repurposed and combined to treat rare diseases. This innovative approach means treatments can be found more quickly, efficiently and cost-effectively than traditional drug discovery methods allow.

Healx was founded in 2014 in Cambridge, UK, by Dr Tim Guilliams, a Biochemical Engineer and Tech Entrepreneur, and Dr David Brown, co-inventor of Viagra and former Global Head of Drug Discovery at Roche. For more information, please visit www.healx.io or follow us on Twitter and LinkedIn.

About Muscular Dystrophy UK
Muscular Dystrophy UK is the charity bringing individuals, families and professionals together to beat muscle-wasting conditions. They offer practical and emotional support for people with a muscle-wasting condition and their families at every stage.

They are working to ensure everyone with a muscle-wasting condition has the right specialist NHS support and healthcare, whatever their age and wherever they live. Muscular Dystrophy UK also supports world-leading research and won’t stop until they find treatments and cures for all our conditions. They help people with all muscle-wasting conditions to improve quality of life, make the most of their independence and ensure every day counts.

For more information or to help support their work, visit www.musculardystrophyuk.org or call the free helpline, Monday to Friday, 10am–2pm, on 0800 652 6352.