DMD research

Women and Duchenne – Dr Alessandra Gaeta, Director of Research

To celebrate World Duchenne Day on 7th September 2022, with this year’s theme of ‘Women in Duchenne’, Duchenne UK is amplifying the voices of inspiring women working in or impacted by Duchenne muscular dystrophy (DMD). Whether they are scientists, doctors, mothers and caregivers or carriers, women are playing a vital role in the fight against DMD, a rare genetic disease that mainly affects boys.   

As Director of Research at Duchenne UK, Dr Alessandra Gaeta leads on the charity’s research portfolio, guiding decisions about where to invest our funds to make the most impact for people living with DMD.   

We sat down with Alessandra to find out more about her career in science and what’s going on at Duchenne UK and in the wider DMD research community. 

Tell us a bit about your career background and how you came to work in the field of DMD? 

“I’ve been working in research and development (R&D) for 20 years, and have a background in industry and the public sector. I completed a PhD in Medicinal Chemistry and previously worked as a research scientist, before moving into research management.  

As a pharmacy graduate, I chose to pursue a career in R&D so that I could ultimately help patients, and working for a patient organisation like Duchenne UK has provided me with the opportunity to have a direct impact on people’s lives and add value.”  

What does your day-to-day role look like?  

Every day is different at Duchenne UK, as we’re working on so many different, exciting projects!  

My role involves identifying new project opportunities that we could become involved with. I have lots of meetings as we collaborate closely with external partners and researchers to share expertise & resources, ensuring that our projects deliver a meaningful impact on the DMD community.  

As well as managing new and current projects, I drive our research strategy, which is informed by the needs of our community and our vision for Duchenne UK as an organisation.”  

What’s going on in the DMD research field at the moment?  

“The DMD field is an exciting area of research; more drugs are in clinical development than ever before, and many of these are not only addressing symptoms but trying to treat the root cause of the disease (for example, gene therapy). We hope to reach the point where these treatments are approved for use in DMD, meaning that more companies will want to invest in the development of this type of therapy for DMD.  

There are also some promising new technologies in pre-clinical development (not yet being tested on humans), such as cell therapies, and treatments that aim to regenerate muscles.  

Another area of research we’re investing in is better understanding of the disease, how it progresses, and how we can develop more robust measures to evaluate the impact of treatments.  

Duchenne UK is also looking at the value that technology can bring to quality of life, and is driving the development of innovative assistive mobility devices that address the unmet needs of people with DMD.  

Our role as a funder and patient organisation is to push for the treatments and technologies that have promise and bring them to the wider community. There’s still lots of work to do, but through collaboration, we are making meaningful progress in the fight against DMD.”  

Which area of research are you most excited about?  

“Although gene therapies have the potential to change the way genetic diseases like DMD are treated, I’m also excited about the impact that technology could have on disabled people’s lives and the role that we as a charity could play in this. For example, by funding the development of the Smart Suit, to help young people with DMD and other neuromuscular diseases with the use of their upper limbs.  

The assistive device market for people living with disabilities is really underserved, and their needs are overlooked. We are working with the people that need these products and other organisations to prove that the development of user-focussed, medical devices can be approached differently.”  

What do you consider to be the highlight of your career so far?  

“It’s hard to pinpoint, there have been many highlights! But working in a patient organisation was a long-term ambition of mine, so it was great to join the Duchenne UK team last year.”  

How can people learn more about the science of DMD and the latest research developments?  

“Duchenne UK and other patient organisations, such as PPMD in the US and the World Duchenne Organization, are working to make DMD science and research more accessible to the DMD community. If you haven’t already, you can sign up to our newsletter to hear more about the research we’re investing in, as well as wider news from DMD research, and follow patient groups on social media.  

We encourage you to get in touch with us if you’d like to find out anything more about the research we’re funding.”  

Find out more about the research Duchenne UK is funding 

World Duchenne Awareness Day is an initiative from the World Duchenne Organization which takes place each year on 7th September.  Find out how to get involved on the WDO website.

Published on 5 September 2022

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