Advanced Therapies Satellite seminar: The Advanced Therapies portfolio at the John Walton Muscular Dystrophy Research Centre

PAST EVENT

We were delighted to welcome Professor Michela Guglieri to deliver our NHIP Advanced Therapies Satellite seminar on 15^th September. Michela described the advanced therapies portfolio at the John Walton Muscular Dystrophy Research Centre, with a specific focus on lessons learned from the team’s increasing expertise in delivery of gene therapy clinical trials and sharing key factors for successful delivery of these trials.

Looking to the future, Michela highlighted the recent publication of a White Paper, sharing recommendations for improved, standardised future practice on the delivery of, as well as referral pathways for, gene therapy clinical trials and licensed products for Duchenne Muscular Dystrophy.

We would like to thank Michela for sharing the team’s experience and recommendations with us.

To access the recording of the seminar please visit here.

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The NHIP Advanced Therapies Satellite is running a series of seminars to highlight key topics and advances in translational and partnership working in Advanced Therapies research and delivery. Professor Michela Guglieri will deliver our next seminar on the topic of the Advanced Therapies portfolio at the John Walton Muscular Dystrophy Research Centre (JWMDRC).

 

Michela obtained her medical degree at Milan University, where she also completed her neurology residency, before moving to Newcastle specifically to develop expertise in translational research and clinical trials. As well as having significant clinical expertise, Michela has a vast amount of experience of running clinical trials in Duchenne Muscular Dystrophy (DMD) and other neuromuscular disorders. Michela is interested in supporting improvement of care and treatment for patients with neuromuscular disorders as well as drug development and translational research, to share the experience from DMD more widely.

 

Michela will give an insight into the Advanced Therapies portfolio at the JWMDRC, based at Newcastle University and Newcastle upon Tyne Hospitals NHS Foundation Trust. The JWMDRC team work towards the development and application of genomic and translational medicine to improve the health outcomes of people living with neuromuscular diseases, consolidating Newcastle’s distinguished, international and world-leading record in research and care in this population.

 

The JWMDRC has an established, and growing, portfolio of advanced therapies clinical trials, offering patients the opportunity to participate in cutting-edge research. The JWMDRC treated the first DMD patient in the UK in a pioneering gene therapy trial and the first patient in Europe with late onset Pompe disease in another gene therapy trial. Michela will give an overview of the advanced therapies research activity at the JWMDRC and lessons learned in the context of rare neuromuscular diseases, as well as providing an insight into how this fits into the wider drug development pathway for these therapies, some of which are now becoming licensed treatments.

 

Join us virtually on Friday 15 September 2023, 10:00-11:00am . To sign up please visit here, details to follow.

 

The following Advanced Therapies Satellite seminar, How the Pan-UK Pharmacy Working Group are optimising ATMP implementation in the UK, presented by Anne Black, Regional QA Specialist Pharmacist for the North East and North Cumbria and Chair of Pan UK Pharmacy Working Group for ATMPs, will take place on the 23 November 2023, 11am – 12pm. Details to follow.