Reverse Rett Matters (February 2022)

February 2022

CONTENTS Welcome Letter 2021: A Year in Review I Am Rare, I Am Registered Talk About Rare Clinical Trial Update Back in North Berwick Inspirational Fundraisers Five in a Row Reverse Rett Statham Link 1F Lancastrian Office Centre Talbot Road, Old Trafford Manchester M32 0FP

Communication is good Where There’s a Will

0161 413 0585 info@reverserett.org.uk www.reverserett.org.uk TRUSTEES Chair: John H Sharpe Vice-Chair: Helen Simmonds STAFF TEAM CEO: Rachael Stevenson Clinical Trial Lead/Supporter Care: Andy Stevenson Finance: Rashida Hossain Events/Rett Connect: Beth Johnsson Design/Marketing: Wesley Havill Patient Registry Co-ordinator: Ewelina Wasowska Fundraising special events: Hazel Beiny Reverse Rett Matters Designer: Wesley Havill Edited by: Rachael Stevenson

The views expressed herein do not necessarily represent the views of the editor or of Reverse Rett Board of Trustees. Parts of this publication may be reproduced with permission, please email rachael@reverserett.org.uk CHANGE IS WITHIN REACH

Hello and welcome to our second Reverse Rett Matters newsletter.

This newsletter has been waylaid by (you guessed it) Covid. Almost everyone on the team has now had Covid, as have many of our family members, with most of us affected over Christmas and New Year and the weeks since.

I’m sure you’ll agree that despite the forthcoming changes to restrictions, things don’t feel quite normal yet but hopefully we will get there. After another disrupted year, we were glad to say goodbye to 2021.

As a patient organisation which runs with no statutory funding, much of our focus at Reverse Rett is necessarily on raising the funds we need to operate and implement our goals. 2021 was another tricky year for us with an extended national lockdown early in the year and a very slow reengagement with in-person events which just started to get going again around July.

I am so thankful to everyone who came out from under the lockdown ready to take on a challenge, despite all the other things that were going on in their lives. You will see from our 2021 in review pages (4 and 5) how, with the help of our stalwart supporters, we were able to pull back our income to almost pre-pandemic levels.

This is a true testament to the commitment and dedication of our supporters and their wider friends and communities.

You can see too, all the things we managed to achieve despite the ongoing disruption and 80% of the team furloughed between January and April 2021.

By year end, despite everything, we’d still met our funding commitment to the CIPP Rett Centre, still successfully recruited for the UK adults’ Anavex trial, still re-developed the Rett Registry UK. We’d still made contributions to the international research projects at the University of Edinburgh and to MECP2 Duplication projects in the USA, albeit at much lower levels than before the pandemic.

On pages 4 and 5 you can read about some of the fundraisers who helped make all this and more happen. Although a lot of the emphasis here is on raising the money we need to achieve our goals, there are so many other ways you can support Reverse Rett too, including getting behind Rare Disease Day and other awareness raising campaigns. If you have a child or other family member with Rett Syndrome, the most important thing you can do to get behind Rett Syndrome research is register on the Rett Registry UK. When we are advocating for improved clinical care and for emerging treatments for Rett Syndrome to come to the UK, every single person with Rett Syndrome matters and needs to be included. Please share the ‘I am rare. I am registered,’ campaign as much as you can.

As this newsletter is coming to you late, we will plan to send the next one out mid April.

We hope to have had further positive news about upcoming gene therapy trials before then. In the meantime, we wish you a healthy and peaceful spring.

All best wishes,

Rachael x

CHANGE IS WITHIN REACH

2021 IN REVIEW

Despite the challenges of lockdown, 2021 was the year that fundraising began to return.

It didn’t get back to the levels we were at before the Pandemic but as restrictions eased in June, organised, mass participation events were back on the agenda.

We are truly grateful to all of our incredible fundraisers and supporters who turn up again and again for our children. Thank you to each and every one of you!

Here are some of the fundraising stories from 2021...

Jamie McDonald braved an Aberdeen January by spending most of it running. He began on NYD by running 1 mile and then adding a mile each day until he ran 31 on January 31st. He was inspired to run like Forrest Gump by his cousin’s daughter, Shona. Jamie ran 507 miles during January and raised £3643.

Jamie

The incredible Vincent Johnsson finished his 500 consecutive 5k runs and celebrated by doing his 501st! He was finally able to throw his running shoes in the bin and reflect on raising £18,950 in honour of his daughter, Hannah. He then handed the baton on to family friend, Gill Sanders who began 365 days of 5k bike rides.

Vincent

Gill and Hannah REVERSE RETT MATTERS

CHANGE IS WITHIN REACH

Former footballer, Mark Reilly took on two incredible challenges in honour of his daughter, Dionne. Starting at St Johnstone's ground in Perth he ran non-stop to Motherwell and St Mirren before finishing at Kilmarnock, a distance of 100 miles.

Mark played for all 4 clubs in a distinguished footballing career. He completed the challenge in 23 hours and it gave him a taste of what was to come later in the year.

The Marathon de Sables is widely regarded as the toughest foot race on the planet. It’s a 156 mile run through the Sahara Desert over 6 days. Mark took on and conquered it despite soaring temperatures and a stomach bug which swept the camp, resulting in a record number of withdrawals. Mark struggled to eat during the week and relied on his legendary grit and determination to make it to the finish line.

Mark

Mark inspired incredible support from family, friends and the wider public raising £18,254.

Despite having Rett Syndrome, Sharna Beeney had always wanted to climb the 02 Arena and finally got her chance last June when she completed the challenge with her mum, Juliette, raising £515.

Sabrina McPeake, her family and friends had a fun time at the Exeter Inflatable 5k. They all had a really great day and are already looking forward to going back in 2022. They were inspired by Sabrina’s daughter, Sienna.

Rita and team REVERSE RETT MATTERS

Sharna

Kicking off in-person fundraising in style, a 20-strong team of Reverse Rett supporters from around the country ran the Asics London 10K in July. The team came together at short notice: London runners included Reverse Rett Co-Founder and Trustee, Rita Ross and all the friends she could rope in at short notice, as well as Reverse Rett Head of Finance, Rashida Hossain, with Reverse Rett team members, Beth Johnsson and Rachael Stevenson and Rachael's sister Sarah Weston, each running locally, at home (but with the London team in spirit!) due to ongoing shielding efforts. All together, this team raised an incredible £10,836!

Ben Hallam had never heard of Rett Syndrome but after reading about Emilia in his local paper he decided he wanted to do something to help. He took on the NC500, a 500 mile adventure on a bike around the Scottish Highlands completing it in 36 hours. Ben raised £5080 and showed how important Rett Syndrome awareness is.

Ben

Beth

After a year of tough, outdoor swimming training, Beth Hosker joined the girls ALIVE relay team and swam the channel. The crossing from Dover to Calais is something every swimmer wants to achieve and Beth raised over £1300 in honour of her daughter, Skye.

Neil Fowler has been chomping at the bit for 5 years, desperate to complete the Ciar Fowler 500, a non-stop bike challenge which he first attempted in 2016. After some serious training he finally did it raising almost £10,000 in honour of his daughter, Ciara. That’s one huge monkey off his back.

Louise Wainwright, mum to Georgia wanted to do something spectacular during Rett Syndrome Awareness Month in October and decided to have her head shaved in public. Louise has always loved her long locks so this was a massive deal for her. She did it in public and alongside a raffle and auction managed to raise £4433

We had 4 very special people running the London Marathon for us this year. Trevor Jones, Laura Palmer, Peter Akrill and Live Aakvik all chose to run for Reverse Rett after gaining entry through the ballot, even though none of them have a family member with Rett Syndrome. The guys all got round safely and raised over £5000 in honour of Georgia, Amber, Martha and Lysbeth.

Although they didn’t finish until 2022 the awesome Five in a Row team set off from the Canaries on their 3000 mile row to Antigua in the Talisker Whisky Challenge on 12th December 2021. They finished just over 36 days later! They were inspired by Eliza, the daughter of crew member, Ross McKinney. Ross, Duncan, Clive, Ian and Fraser got 2022 off to a great start by raising £49,608!

REVERSE RETT MATTERS

Five in a Row CHANGE IS WITHIN REACH

20 REVERSE RETT 21 IN REVIEW GROSS INCOME

792,485 PERCENTAGE INCREASE ON 2020

UK CLINICAL TRIALS OF POTENTIAL NEW TREATMENTS AT UK TRIAL SITES

SOURCES OF INCOME

DONATIONS ONLINE GIVING EVENTS TRUSTS & FOUNDATIONS

PHARMA GIFT AID COVID GRANTS

THERE'S ALSO ANOTHER TRIAL DUE TO START IN 2022 WITH UP TO 6 TRIAL SITES

AFTER A DRAMATIC DIP IN INCOME IN 2020; IN 2021, WE REINVESTED IN LIVE EVENTS TO BRING OUR FUNDRAISING BACK ON TRACK.

GROSS EXPENDITURE

849,486 HOW WE SPENT RAISED FUNDS

CHARITABLE ACTIVITIES FUNDRAISING COSTS MANAGEMENT

IMPROVED INCOME MEANT WE COULD INCREASE RESEARCH FUNDING, ALTHOUGH STILL NOT BACK TO PRE PANDEMIC LEVELS.

Taking part in a clinical trial has given us hope for treatments to help alleviate some of the symptoms of Rett.

I thought it would be really stressful to organise everything for the study visits, but Reverse Rett took that stress away by making all our arrangements including travel and an accessible hotel. It really did take a great weight off my mind."

- Mum to C

RESEARCH FUNDING INCREASE ON 2020 IS UP BY

2021 RESEARCH FUNDING 430,320

Reverse Rett are a lifeline. Having a daughter with Rett Syndrome is tough but the energy and enthusiasm Reverse Rett brings to fundraising and raising the profile of this syndrome - inspires lots of hope. They are always on the end of the phone if anything crops up (which is very common living in this unpredictable world of Rett Syndrome). Great charity run by great people.

- Victoria

CIPP RETT CENTRE CIPP RETT MONITORING RETT LABORATORY RESEARCH MECP2 LABORATORY RESEARCH

HOW WE'RE BUILDING BACK TO PRE-PANDEMIC LEVELS PROJECTIONS FOR 2022 RESEARCH FUNDING

545,045

2021 OUTREACH CIPP RETT CENTRE CIPP RETT MONITORING RETT LABORATORY RESEARCH MECP2 LABORATORY RESEARCH

BITESIZE SESSIONS WITH OVER ATTENDEES

PROJECTED % INCREASE FOR 2022

FAMILY FORUM SESSIONS WITH 10-30 ATTENDEES

RETT REGISTRY UK

UP CL SE 503 PATIENTS 78 NEW ENTRIES 15.5% INCREASE

ENGLAND SCOTLAND NORTHERN IRELAND WALES

FEMALE 97% MALE 3% 182 ADULTS 321 CHILDREN

30 PATIENTS REFERRED TO CLINICAL TRIALS

388 UK 59 EUROPE 56 INTERNATIONAL

152 PATIENTS MONITORED THROUGH CIPP RETT DATABASE

135

NEW SUBSCRIBERS

THE

REVERSE

RETT PODCAST

DOWNLOADS

WWW.YOUTUBE.COM/REVERSERETTUK

If you have any reservations about joining the Family Forum, please be assured you won’t be disappointed and you will come away feeling so much stronger and armed with knowledge for your child.

- Helen

This Rare Disease Day, help Reverse Rett by sharing the ‘I am rare. I am registered,’ campaign so that together, we can make sure everyone with Rett in the UK is counted.

Rare Disease Day 2022 will be held on February 28th.

At Reverse Rett, this Rare Disease Day, we are focusing our efforts on stressing how important it is that everyone with Rett Syndrome in the UK, is registered on the Rett Registry UK. At Reverse Rett, we are doing a lot of work to advocate for clinical trials and new treatments to come to the UK, but we need to have accurate and up to date information about how many people in the UK have Rett Syndrome and updated information on their status.

When we are doing this work, we can only count your child, young person or adult with Rett Syndrome in, if they are registered on the Rett Registry UK.

Hannah

28th February 2022

#RareDiseaseDay #RettRegistryUK We need to show industry partners, regulators and payers in the UK and beyond that everyone with Rett Syndrome in the UK counts and each and every one matters.

Help us, by ensuring that you’re registered and by sharing your own, ‘I am rare. I am registered,’ post on social media on February 28th 2022.

Ahead of the big day, you can also help us develop infographics to use around Rare Disease Day to help promote the ‘I am rare. I am registered,’ campaign.

Simply send us an image of your child, adult or young person with Rett or MECP2 Duplication Syndrome to use and complete this sentence: I am rare because….

I am registered because…..

REVERSE RETT MATTERS

CHANGE IS WITHIN REACH

TALK ABOUT RARE! Young people are some of our most creative and effective fundraisers, with support for Reverse Rett from schools proving hugely successful over the years.

However, like many things, school fundraising was largely put on hold by Covid19, with ‘mixed bubble’ events like fetes, sales, concerts and assemblies all paused.

But with school events now creeping back into the calendar, there’s never been a better time to get schools involved, and Rare Disease Day on Monday 28th February is a great place to start!

Many schools now have Charity Reps who are keen to hear from new charities and to nominate smaller charities for their fundraising ventures, especially those with a personal connection to the school.

Why not ask if you (or your child/children) can say a few words about Rett Syndrome to mark Rare Disease Day?

We’ve even made a simple presentation, which we’ll customise for you, to help!

Watch an example presentation here and get in touch with wesley@reverserett.org.uk to have it customised just for you :)

Rett Syndrome Andy's section

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UK CLINICAL TRIAL UPDATE FEBRUARY 2022 The Anavex 2-73 clinical trial (in adults 18 and over), began last year. It is taking place at the CIPP Rett Centre at King’s College Hospital in London, under Professor Santosh and the CRF at St Mary’s Hospital, Manchester under Dr Banka.

The trial has been completed in the United States with no adverse result or safety concerns. We are currently waiting for top line data for the drug to be released.

CIPP RETT CENTRE

The Anavex 2-73 clinical trial (in children between 5 and 17) is due to start imminently. We are waiting for confirmation of the trial sites (there could be as many as six in the UK) and we hope to start pre-screening calls with parents and caregivers soon. The trial has been delayed but everything is still going to plan. We had an overwhelming response to our Expression of Interest email which was sent to eligible patients in September 2021 and we are looking forward to the trial getting underway.

As far as clinical trials go this one is quite straightforward to take part in. It is 7 weeks long with an optional 12 week extension period. There are no overnight stays and a maximum of 4 in-person hospital visits. Anavex are hoping for a global improvement in symptoms as the primary outcome measure The best way to access UK clinical trials for Rett Syndrome is by registering your child or adult with Rett Syndrome on the Rett Registry UK. Reverse Rett has provided clinical trial support service to all UK Rett trials to date.

Taysha Gene Therapies are still on track to begin gene therapy clinical trials at trial sites around the world during 2022.

Click here to join the Rett Registry UK rett registry uk

REVERSE RETT MATTERS

8

CHANGE IS WITHIN REACH

BACK IN NORTH BERWICK! After two years away from Scotland, we are delighted to be returning to North Berwick this May for our fifth Reverse Rett Run at Archerfield Walled Garden, sponsored by Paris Steele.

Runners of all ages and abilities can take on the 10k or 5k route, whilst the whole family can enjoy stalls, activities, entertainment and the kids’ fun-run, all within the beautiful Walled Garden grounds.

This is always a wonderful event, bringing together an amazing local community as well as families and runners from further afield for a day of awareness-raising, friendshipbuilding and, of course, crucial fundraising!

Registration for the 10k and 5k races is now open, with a discount for anyone setting up a JustGiving page to gather sponsorship for their efforts!

Registration for the kids’ 1k fun-run will take place on the day and all participants will receive a biscuit medal to decorate!

If you don’t fancy running but would like to get involved, we are always in need of volunteers to help make the day run smoothly - please get in touch with beth@reverserett.org.uk We’re so excited to be going back to North Berwick and very much hope to see you there!

1.

Click here to sign up 2.

REVERSE RETT MATTERS

CHANGE IS WITHIN REACH

INSPIRATIONAL FUNDRAISERS

FIVE IN A ROW When Ross McKinney’s North Berwick Coastal Rowing team mates, Duncan Hughes, Clive Rooney and Ian Baird told him they were going to row the Atlantic in the Talisker Whisky Challenge for Reverse Rett he was delighted. They then asked him to join the team and after discussing the challenge with wife, Catherine, he and Fraser Potter joined and ‘Five in a Row’ began the lengthy process of getting ready for one of the toughest challenges in the world.

Although they began the race on December 12th 2021 the preparation started two years earlier. Firstly, there was a boat and £120,000 of corporate sponsorship to find before they could even consider racing. This was no easy task especially in the middle of a Pandemic. There were all kinds of courses that needed to be passed to ensure the team could find their way across the Atlantic and stay safe while doing it. They also had sessions with a psychologist as this challenge was as much mental as physical and there was also the fact that they needed to get in to shape to take on such a brutal, physical challenge.

The first week they all suffered from sea sickness and the pattern of rowing for 2 hours on, 2 hours off, 24 hours a day was a real shock to the system.

With all of these obstacles overcome they headed out to La Gomera in the Canaries in early December to prepare for the 6 weeks ahead. They weren’t the only ones preparing, the boys were leaving their families for around 8 weeks in total and they would have to come to terms with them missing Christmas and New Year. Not easy, especially for Catherine who had Eliza with Rett Syndrome and three other children to look after. This was not something that could be undertaken without the full support of every family member.

Then they were off. They knew what to expect, they’d done the training, spent plenty of time on the boat, they couldn’t be more prepared. But knowing and doing are two different things. The first week they all suffered from sea sickness and the pattern of rowing for 2 hours on, 2 hours off, 24 hours a day was a real shock to the system. The heat was brutal and the cabins tiny and smelly. This was no holiday! They also had to clean the hull of the boat each week which meant diving in and scrubbing it, it must have made a change from rowing until one of the boys saw a shark circling close by!

CHANGE IS WITHIN REACH

REVERSE RETT MATTERS

As the days wore on, the blisters got bigger and the bums got sorer but they persevered even through some storms with 40ft waves which soaked them continually and even threw them from their seats.

Finally after 36 days, 3 hours and 33 minutes and 3000 miles rowed, Five in a Row made it to Antigua and the finish line to be greeted by their families. They also finished in 3rd place after a fierce battle with the Atlantic Flyers team who pipped them to 2nd spot. What an incredible achievement!

They have raised an almighty £49,248 with donations still coming in. What a difference that will make to the work we do at Reverse Rett.

CHANGE IS WITHIN REACH

You can support Five in a Row by visiting their Just Giving page here

Huge thank you to Duncan Hughes, Clive Rooney, Ian Baird, Fraser Potter and our very own, Ross McKinney for choosing to support Reverse Rett, in honour of Eliza, on this epic adventure across the Atlantic. You are an inspiration to us all and your efforts will motivate people to get out of their comfort zone and push themselves that little bit further to keep Rett Syndrome research moving forward.

Boys, we salute you!

CHANGE IS WITHIN REACH

COMMUNICATION IS GOOD

REVERSE RETT MATTERS

CHANGE IS WITHIN REACH

My granddaughter who is almost 13, has Rett Syndrome.

As many of you will know Reverse Rett is a small charity and we have to fight for every penny in order to help deliver treatments for the condition.

One of the methods I employ to fundraise is to introduce the charity to those who control trust funds and legacies.

I have been fortunate over the years to have come across sympathetic people who have donated funds from trusts and legacies.

It occurred to me that some of you may, even unknowingly, have friends, relatives or know people who can help Reverse Rett in this way as I have done.

This has only come about by talking. Most grandparents love to show off their grandchildren and talk about them and show pictures. I do this at every opportunity and always try to explain about Rett Syndrome and the crucial work undertaken by Reverse Rett.

We owe it to our girls to explore every possible avenue to raise funds.

This not only raises awareness, but it’s surprising how people can come out of the woodwork who have access to funds available for charities.

This also applies to wills and legacies. There are many people who don’t have relatives to bequest or others who are fortunate enough to have sufficient funds to leave their money to charities as well as their families.

We owe it to our girls to explore every possible avenue to raise funds.

Communication is good - so please talk to people about Rett and enquire whether they know anyone who controls trust funds or legacies.

Thank you

CHANGE IS WITHIN REACH

WHERE THERE’S A WILL . . .

Everyone needs a Will. Whatever your age, whatever your health, whatever your wealth: your Will is your chance to impact the future of those you care about and those yet to come.

Of course, working to impact the future of those you love is something our supporters (you!) are pretty familiar with! Bike rides, golf-days, ocean-rows, quiz nights, desert hikes, bake-sales, firework-bonanzas, live gigs, Channel-swims, baked-bean-baths - we name it, you’ve done it, driven forward by the hope of a brighter future for someone you love.

The good news is that writing a Will doesn’t require any of the grit, determination, blood, sweat, tears, lunacy or baking-skills of the above!

It also doesn’t have to cost you anything now, or cost anyone you love anything in the future.

We’ve partnered with the National Free Wills Network (NFWN) to be able to offer you the chance to write (or amend) your simple Will for free.

Of course, we hope that you’d then consider leaving a gift in your Will to Reverse Rett.

Which doesn’t mean leaving less to the people you love; you can leave a % of whatever is left once your loved ones are all provided for.

Any percentage, no matter how small, could help to make a lasting difference for those living with Rett Syndrome now and for generations yet to be born.

We recently made our will through the National Free Wills Network . It was completely free to do. We chose to make a gift to Reverse Rett because we want all the good work to continue after we’re gone.

- Rachael & Andy

If you'd like a #MakingChangeHappen money box to save up your pennies for this year's Christmas Challenge then please email ewelina@reverserett.org.uk

Reverse Rett Statham Link 1F Lancastrian Office Centre, Talbot Road Old Trafford, Manchester M32 0FP 0161 413 0585 info@reverserett.org.uk www.reverserett.org.uk

Find us on Facebook, Twitter, Instagram and Vimeo under 'Reverse Rett'

Registered charity in England & Wales, charity number 1136809 and in Scotland, charity number SC046735.

A company limited by guarantee number registered in England & Wales, company number 07278507.