GNE Myopathy UK

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GNE Myopathy UK This page is for individuals living in the UK impacted by GNE Myopathy (GNEM). Advocates and caregiv What is the purpose of this group?

This group is for individuals living in the UK who have GNE Myopathy (GNEM). However, those people affected or impacted by GNEM, through being a caregiver, friend, clinician and/or advocate are very welcome to join the group. The objectives of this group are to:

• Provide support to those living with GNEM in the U.K.
• Develop a proactive patient network community sharing knowledge and raising awareness about GNEM;
• Share and develop resources and information about GNEM; &
• Empower patients and caregivers nationally (perhaps even internationally) enabling us to build a more patient-centred healthcare system. We trust all members to treat any information shared in the group with respect and sensitivity. Thank you for your support. What is GNE Myopathy (GNEM)? GNEM is a rare inherited disease found in populations worldwide. GNEM is also known as ‘Hereditary Inclusion Body Myopathy’ (HIBM), ‘Quadriceps Sparing Myopathy’ or ‘Nonaka Distal Myopathy’. GNEM causes severe muscle weakness in the legs and arms, usually leading to physical disability within twenty years of onset. Although, some patients are able to live using just a walking stick for many years, in most patients the quadriceps muscles are spared. GNEM does not affect the vital organs such as the heart and the lungs. For further info, please visit the:

~ Neuromuscular Disease Foundation’s website:
http://curehibm.org/for-patients/

~ Muscular Dystrophy UK (MDUK) website: https://www.musculardystrophyuk.org/conditions/gne-myopathy-gnem

🧵Adaptive clothing opportunity for our rare disease community🧵I’ve recently been contacted by Sambhav Apparels, an India...
27/09/2026

🧵Adaptive clothing opportunity for our rare disease community🧵

I’ve recently been contacted by Sambhav Apparels, an India-based company developing adaptive clothing for disabled people, including those living with progressive conditions and limited mobility.

Their aim is to make everyday and occasion wear easier to put on, practical and affordable, using features such as side openings, Velcro and magnetic fastenings.

They’re looking for disabled people to try an adaptive outfit, customised around their individual needs, and share honest feedback about their experience.

The outfit would be gifted, and in return they’re asking for:

📸 A photo wearing the outfit.
🎥 A short review video of under 2 minutes.
📝 Honest feedback on how practical and easy the clothing is to wear.

They offer both Western and traditional clothing. The review would normally be requested within a week of receiving the outfit, although they understand that circumstances can sometimes cause delays.

🌐 You can find out more about Sambhav and their adaptive clothing here:
https://sambhav.co/

I think this sounds like a really worthwhile project. The more disabled people with lived experience who can share honest feedback, the greater the opportunity to help shape clothing that genuinely makes dressing easier and supports greater independence. 💜

If you, or someone you know, would be interested in taking part, please contact Sambhav Apparels directly on +91 7230002829.

Just to be transparent, I’m sharing this opportunity with our community but haven’t personally tested or reviewed Sambhav’s clothing.






Sambhav designs adaptive clothing for wheelchair users, muscular dystrophy, cerebral palsy and elderly people. Easy-wear kurtas, sarees and shirts with magnetic buttons and velcro.

04/09/2026

🔬GNEM research opportunity: UX016 first-in-human study now recruiting

Some encouraging GNEM research news to share!

The Ultragenyx UX016 clinical study is now recruiting people with GNEM at a research site in Iselin, New Jersey, USA.

Please note that not everyone with GNEM will be eligible. Participants must meet specific study criteria and pass a screening process. This includes requirements around age, confirmed GNEM diagnosis, mobility and muscle function, as well as being able to attend study visits in the US. There are also additional medical and study-specific criteria.

👉 If you think you might be eligible, please contact the study site directly. The research team can explain the full eligibility criteria, screening process and what taking part would involve.

🔗 Study details: https://clinicaltrials.gov/study/NCT07511556

It’s really positive to see new research and potential treatments being explored for GNEM. 🙏

21/08/2026

Get involved and help raise awareness about muscle wasting conditions!

Muscular Dystrophy Awareness Month is only two weeks away.

Across September, we'll spread the word about muscle wasting conditions, how they impact people and the incredible work being done to research and treat them.

But we need your help - here's how you can take part.

1. Post to raise awareness 📢
Share your experiences and spread the word about muscle wasting conditions - and be sure to use the hashtag

2. Fundraise for us 🌟
If you’ve been thinking about fundraising, September’s the perfect time. From running challenges to bake sales, you can fundraise your way.

Get involved: https://loom.ly/xuiVYlc

3. Share your story 💬
Tell us about your journey - whether you live with a condition, or have a loved one who does.

Find out more here: https://loom.ly/JS3XBGo

4. Stay tuned on our socials 🧡
Throughout September, we’ll be highlighting stories from our community, sharing research updates and raising awareness about a range of conditions.

Keep an eye out across Facebook, Instagram, LinkedIn and YouTube.

Go Kam! 📖
21/08/2026

Go Kam! 📖

21/08/2026

Hi friends,

The Neuromuscular Disease Foundation (NDF) is reviewing its website to make sure it’s accessible, inclusive, easy to navigate and meets the needs of our global GNE community. 🌎

We’d really value your input! The survey takes just 3–5 minutes, and your feedback will help shape future improvements.

📋 Survey: https://forms.cloud.microsoft/pages/responsepage.aspx?id=UPs_KAujjEiQ9M2uT3rm0fsMqdektZtFkashFABSE6ZUOEtYOTQ4NkVDQ1RLNUo1NVJHSkpaU1lWUy4u&route=shorturl

📅 Closing date: 31 August 2026

Thank you so much for taking the time to share your views. Every response will help us make the website better for our community! 🙏

We’re incredibly proud of our wonderful community member, Tahira, for sharing her personal story through Muscular Dystro...
27/07/2026

We’re incredibly proud of our wonderful community member, Tahira, for sharing her personal story through Muscular Dystrophy UK and helping to raise awareness of GNEM as part of South Asian Heritage Month. 🧡

In her post, Tahira reflects on the unique challenges of living at the intersection of disability, ethnicity and identity as a British South Asian woman. Her honesty and courage shine a light on experiences that are too often overlooked, while helping others feel seen and understood.

Please take a moment to read and support her Insta post here:
https://www.instagram.com/p/DbS61i6EzFw/?igsh=MWsxa2h3YXR1eGV2NQ==

Thank you, Tahira, for using your voice to educate, inspire and advocate for our community. We couldn’t be prouder. 🫶 👏

07/05/2026
08/04/2026

Hi everyone

Hope you’re managing to enjoy some of these glorious rays that were (very randomly) experiencing in the UK. ☀️

Just wanted to let you know that the Ultragenyx trial is now listed on the official gov site. It’s not yet recruiting though.

31/03/2026

Hi everyone,

Some promising news to share with the community!

💗 Ultragenyx Pharmaceutical has received FDA clearance to begin a clinical trial for a potential new treatment for GNE myopathy (called UX016). 💚

What this means in simple terms:
• The treatment can now move into human trials (Phase 1/2)
• The study is expected to start later in 2026
• It will look at safety first, but also whether it can help muscle function

This treatment is designed to help the body better deliver sialic acid to muscles, which is something people with GNE myopathy struggle to produce.

It’s still early days, and there’s no guarantee yet, but this is an important step forward, especially as there are currently no approved treatments.

Also worth noting: this work is being supported through a patient-led funding approach, which shows the power of our community in driving research forward 💪🏽

As always, I’ll share updates as things progress.

What are your thoughts on this?

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