CDKL5 UK

29/09/2026

Today, we’re sharing the recording of our second webinar in the 2026 From Science to Hope: Walking the Path Towards CDKL5 Gene Therapy series. 💜

Co-produced by the Global CDKL5 Community, this thoughtful discussion explores the important—and often complex—considerations families may face when thinking about disease-modifying treatments such as gene therapy.
Together, speakers discuss topics including:
* Consent and ethics
* Personal choice and uncertainty
* Informed decision-making
* Navigating potential treatment decisions as a family
We are also proud to share that this webinar has been translated into 10 languages, helping make this information more accessible to CDKL5 families around the world. 🌍✨

A heartfelt thank-you to the members of our global community whose time, voices, and expertise made these translations possible. Your efforts help ensure that more families can access information, join the conversation, and feel supported as they explore the questions and possibilities ahead.

Watch the recording, access translations, and find easy-to-read summaries through the link below:
https://youtu.be/V1i9BlD8uBc

20/09/2026

Such sad news. We will be supporting Brynn and her family tomorrow, along with our wider CDKL5 community 💚💚

💚 In Loving Memory of Brynn 💚

It is with profound sadness that we share the heartbreaking news that Brynn Ladly has passed away.

Brynn was a beautiful CDKL5 warrior, and a cherished daughter, whose life touched so many within our CDKL5 community. Her family, Natalie and her loved ones, have given so much to our community over the years, including through Natalie's dedicated service on the CDKL5 Canada Board.

There are simply no words that can adequately express the sorrow of losing a child.

Our hearts are with Natalie and her family as they navigate this unimaginable loss. We hold them close in our thoughts and surround them with the love and strength of the CDKL5 community. 💚

Brynn's school has invited their community to wear green on Monday, September 21, in her honour. CDKL5 Canada would like to extend that invitation to our entire community.

💚 On Monday, September 21, please join us in wearing green for Brynn.

Wear green.
Share a photo.
Light up your home in green.
Or simply carry Brynn in your heart.

For a community that understands how deeply these children are loved, we know that sometimes the smallest gestures can carry enormous meaning.

Today, we remember Brynn.
We honour her life.
We hold her family in our hearts.
And we stand together in love and solidarity. 💚

Please, if you can, give your children an extra hug and kiss today. Hold them a little closer. And for those who knew and loved Brynn, may the memories of her beautiful life bring comfort in the days ahead.

Forever a CDKL5 warrior. Forever loved. Forever part of our community. 💚

With deepest sympathy,
CDKL5 Canada

18/09/2026

Every child with CDKL5 is unique, and the care and support they receive should reflect that. 💜

In partnership with Emma Claydon, our CDKL5 UK-funded Research and Care Coordinator, we recently delivered a bespoke training workshop for the new care team supporting a very special little girl and her family.

Our training is centred around the individual, recognising that each child’s genetic variant, symptoms, presentation and care needs can be different. We take the time to understand the child, listen to their family and tailor our training to ensure care teams have the knowledge, skills and confidence to provide truly personalised support.

Because understanding CDKL5 is important, but understanding the person behind the diagnosis is what really matters. 💜

If you would like to explore this type of support for your child or care team, please get in touch via WhatsApp or email us at [email protected]

16/09/2026

We are all set up at the British Association for Community Child Health Annual Conference for next two days hoping to spread some awareness of CDKL5

12/09/2026

Today at 3pm Join us

📢 DON'T MISS the CDKL5 Global Webinar!

📌 September 12, 2026, 2:00 PM UTC (3pm GMT)

What does informed decision-making look like when considering an emerging therapy? Join us for this important conversation on hope, uncertainty, risks, benefits, and the questions families may face along the way.
We’re excited to invite CDKL5 families and caregivers to our next virtual webinar and panel discussion,“🧬Gene Therapy: Making an Informed Decision : What Parents and Caregivers Need to Consider.”

This webinar will feature Dr. Matthew McCoy, Associate Professor of Medical Ethics and Health Policy at the University of Pennsylvania, and Luke Rosen, a parent who has experienced the complex decisions and considerations within his own family. This discussion will explore:

Balancing hope and realistic expectations
Defining best interests and meaningful benefit
Weighing risks, benefits, and uncertainty

HELP SHAPE THE CONVERSATION BY SUBMITTING YOUR QUESTIONS IN ADVANCE 🔗https://forms.gle/vbcSHpVwX8vu4X4f6

REGISTER HERE if you are not yet https://us06web.zoom.us/webinar/register/WN_6NanWVIeQfu9Bfl5qXTwJw

This is the second webinar in our three-part 2026 series, From Science to Hope: Walking the Path Towards CDKL5 Gene Therapy, co-produced by the Global CDKL5 Community.

01/09/2026

📢 DON'T MISS the CDKL5 Global Webinar!

📌 September 12, 2026, 2:00 PM UTC (3pm GMT)

What does informed decision-making look like when considering an emerging therapy? Join us for this important conversation on hope, uncertainty, risks, benefits, and the questions families may face along the way.
We’re excited to invite CDKL5 families and caregivers to our next virtual webinar and panel discussion,“🧬Gene Therapy: Making an Informed Decision : What Parents and Caregivers Need to Consider.”

This webinar will feature Dr. Matthew McCoy, Associate Professor of Medical Ethics and Health Policy at the University of Pennsylvania, and Luke Rosen, a parent who has experienced the complex decisions and considerations within his own family. This discussion will explore:

Balancing hope and realistic expectations
Defining best interests and meaningful benefit
Weighing risks, benefits, and uncertainty

HELP SHAPE THE CONVERSATION BY SUBMITTING YOUR QUESTIONS IN ADVANCE 🔗https://forms.gle/vbcSHpVwX8vu4X4f6

REGISTER HERE if you are not yet https://us06web.zoom.us/webinar/register/WN_6NanWVIeQfu9Bfl5qXTwJw

This is the second webinar in our three-part 2026 series, From Science to Hope: Walking the Path Towards CDKL5 Gene Therapy, co-produced by the Global CDKL5 Community.

28/08/2026

Your voice matters in decisions about access to medicines. 💜

We’re sharing this invitation for a webinar that will explain how Health Technology Assessment (HTA) works and how patients and caregivers can help ensure the CDKL5 community’s experiences are heard.

HTA is part of the process used to assess which medicines may be available through public healthcare systems. It is not the same as regulatory drug approval, but patient and caregiver perspectives can play an important role in decisions about access.

📅 Saturday 19 September 2026
🕐 13:00–14:30 BST | 14:00–15:30 CET
💻 Join on Zoom: https://us06web.zoom.us/j/82789148636?pwd=WWPi52Ix84Hab81CcTGBKcd10bqzh5.1

The 90-minute session includes:
• 60 minutes exploring the basics of HTA, how it works, questions families may be asked, common ways to get involved, and tips for sharing useful information
• 30 minutes of discussion about what HTA means for the CDKL5 community

Presented and moderated by Neil Bertelsen, an expert in patient and caregiver involvement. The webinar will be recorded for anyone unable to attend.

The webinar will be conducted in English, with automatic translation subtitles available in other languages. Subtitles work better in the Zoom app, which you can download at https://zoom.us/download. You can also join through the Zoom website.

Please share this invitation with other CDKL5 families. Together, we can make sure lived experience is part of the conversation. ✨

Special thanks to UCB for funding and supporting the creation of this webinar. UCB had no role in its content, which was developed by Neil Bertelsen following interviews with key members of the CDKL5 community.

17/08/2026

Registration Open - September 12 - 2pm UTC (3pm BST)

We’re excited to invite CDKL5 families and caregivers to our next virtual webinar and panel discussion:

Gene Therapy: Making an Informed Decision : What Parents and Caregivers Need to Consider

Presented by the Global CDKL5 Community, this webinar will explore some of the important and often complex considerations families may face when thinking about a disease-modifying treatment such as gene therapy - including consent, ethics, choice, uncertainty and informed decision-making.

This is the second webinar in our three-part 2026 series, From Science to Hope: Walking the Path Towards CDKL5 Gene Therapy, co-produced by the Global CDKL5 Community, as as before there will be translation options, the opportunity to submit questions in advance, and the recording will be available to registered attendees

We look forward to welcoming you again as, together, we explore how families can navigate these complex and deeply personal decisions with the information they need.

You can register here :
https://us06web.zoom.us/webinar/register/WN_6NanWVIeQfu9Bfl5qXTwJw #/registration

And stay tuned for the final webinar in our series: a closer look at what taking part in a clinical trial may involve.

28/07/2026

check it out

We’re excited to share two new videos now available on the IFCR YouTube channel, recorded at our 2026 CDKL5 Family Educational Conference. These sessions offer key insights on gene therapy and what it could mean for the CDD community. 💚

▶️ ELJ‑101: AAV Gene Therapy for CDKL5 Deficiency Disorder
An update on ELJ‑101, an investigational gene therapy from Elaaj Bio, including what families should know today. This video is for educational and informational purposes only. Learn more at Elaaj Bio’s FAQ page: https://www.elaajbio.com/

▶️ Gene Therapy Trials: Hope and Honest Conversations
A clear, balanced look at how gene therapy works, what current trials involve, and the potential benefits and risks for families.

Watch both sessions now on our YouTube channel and continue learning with us✨

26/07/2026

🗓️ Mark your calendars! We’re so excited to invite you to our upcoming CDKL5 Support Workshops.

We know how busy life can be, especially when balancing work, care, and everything in between. That’s why we’ve scheduled these sessions for the evening: so you can "dip in" when the day has settled down. These workshops are designed to be a safe, supportive space for our families to connect and learn together. We’ll be sharing more about our other topics: Managing Uncertainty, Supporting Siblings, and Managing Transition: for the Spring soon!

Save these dates:

💜 Tuesday 13th October, 7pm - 8.30pm
"Living with a rare condition: you and your emotional wellbeing"
A space to acknowledge the emotional load and focus on your own resilience and wellness.

💜 Tuesday 10th November, 7pm - 8.30pm
"Dealing with Diagnosis"
Whether your diagnosis is new or you’re still navigating its impact, we’re here to walk through it with you.

We are also working closely with other organisations to bring you even more vital information. We’ll soon be releasing dates for additional workshops covering:

🧬 Gene Therapy: What’s on the horizon?
📊 Health Technology Assessments (HTA): Understanding the process and how you can get involved to shape the landscape of CDKL5 research.

We’re here to empower our community with the knowledge and support you deserve. You are not alone on this journey, and your voice matters.

How to get involved:
💬 Message us here on Messenger
📱 Reach out via WhatsApp
📧 Email: [email protected]

We can't wait to see you there!