Re-emerging from lockdown

by Florence, National Director

The past 18 months in lockdown have been undoubtedly difficult for most and devastating for many. What has come out of this exceptional time in history, however, is the power of humanity. The times we have pulled together, to help others, to support those who are less fortunate and to think of the greater good in the world. 

A shining example can be seen in the ‘Captain Tom 100’ challenge that many of you undertook this month, as we came together to celebrate the life of such a wonderful gentleman.

A big thank you to our ‘Captain Tom 100 Heroes’; Mary Down who held a plant and cake sale, Sign Solutions who took on a sign language challenge and Dougie Young who lives with Duchenne who drew 100 pictures! Together they raised over £1,700, a phenomenal amount of money, which will make a significant impact.  We are grateful not only to each of these wonderful people, but also to everyone who got on board and donated to their fundraising pages. 

Start small, think big!

The Captain Tom Heroes are testament to the power of coming together, starting small and working together to think BIG! With this in mind, this week, we have launched a brand new (and FREE!) fundraising pack to help you get started. You simply pay the postage cost and we will send you a parcel of goodies that you can use to get your fundraising underway! As always, our Fundraising team are always here to help if you need ideas and inspiration, simply get in touch.

New transition project

This week, I am thrilled to announce our new partnership with the wonderful and truly inspiring Demelza Hospice Care for Children.  Demelza have selected us to deliver their ground-breaking transition to adulthood project, and this week we are kicking off with a fact-finding survey. We will be finding out what young people want and need, versus what clinics provide. Based on our findings, we will work with Demelza to find out the shortfalls and in the longer term aim to deliver an all encompassing support system. If you are a young person or adult living with Duchenne, now is your chance to have your say and play a part in shaping the future.

Translarna

We have been working in partnership with MDUK and families living with Duchenne to campaign for access to Translarna, and have been representing the Duchenne community on the Managed Access Oversight Committee since the Managed Access Agreement (MAA) for Translarna began in 2016.

This week we have shared the welcome news that the MAA for Translarna is to be extended from July 2021 to January 2023, which will be a relief to the families who were waiting for confirmation. We also brought you the news that the Scottish Medicines Consortium accepted Translarna for use on NHSScotland for three years through the ‘ultra-orphan pathway’. More information can be read in our news story here

We will continue to represent patients, families and the community and are here to support you. Should you have any questions please contact our Head of Research Neil Bennett neil@actionduchenne.org.

Science on Tour

Let’s get this show on the road! We have been busy planning the series of Science on Tour events and getting the interactive activities built, ready for the launch in June! Our outreach showed that what Duchenne families really want is more information about the recent developments in science. 

The Science on Tour events do just what they say, we’re bringing easy to understand Duchenne science to you! Each of the 24 events across the UK will be pandemic dependent, but we hope that since they are designed to be small, they will be possible. The Duchenne community has jumped at the chance to learn more, as sign-ups have been swift.  Some of the events are fully booked now, with waiting lists in place, so if you haven’t already, please do sign up to secure your place. 

Supporting you

As always, we are here to support you and your family. As the UK starts to open up, you may find you are struggling with expectations, returning to ‘normality’, resuming regular schooling, seeing family and friends. If you are feeling overwhelmed, you are not alone. 

We are supporting many Duchenne families at the moment who are finding life really tough. And we’d like to help you too. 

Please drop us an email or send us a request for support and we’ll get in touch with you.

Until next time, take great care and best wishes, 

Florence