Have you discovered our latest edition—RARE and social, sponsored by @PTCBio? This edition focuses on the good and bad of social media in rare disease advocacy. Inside this edition: - Under the Same Sun grassroots social media campaign, changing narratives for people with

✨ It’s Day Five of our 24 Days of RARE Inspirations ✨ Introducing Suvam Bhatta, a rare disease patient advocate and disability rights advocate living with limb-girdle muscular dystrophy (LGMD). 💬 Here’s what was said about Suvam: “Suvam is a young disability and rare

BridgeBio's FORTIFY phase 3 study is a turning point for limb-girdle muscular dystrophy (LGMD) patients. Chief Medical Officer Dr. Doug Sproule shares the promising results bringing real hope for new therapies and inspiring innovation across the rare neuromuscular disease

✨ Welcome to Day Four of our 24 Days of RARE Inspirations ✨ Introducing Sierra Domb, who lives with three rare conditions: Erythromelalgia (EM), a rare neurovascular pain disorder, Autoimmune Dysregulation, and Visual Snow Syndrome (VSS). She is also the founder of the Visual

We asked our community "Do you feel the quality-of-life assessment form adequately allowed you to convey the full impact of your condition, or that of your loved one, and what a good quality of life means to you, or your loved one?". insights brings you the latest views from your

Step into the world of Vicky, a RARE parent, as she shares an intimate and inspiring insight into raising her daughter, Tiger-Lily, who lives with congenital central hypoventilation syndrome (CCHS). From daily routines and navigating medical challenges to finding immense joy and

BBC Radio 4 listeners raised £34,057 for vital research into rare brain diseases PSP & CBD, doubling donations through match-funding. The funds will support six new UK studies, bringing hope to affected families. https://t.co/KOYxOeEcyy #BBCRadio4 #Fundraising #PSPA #RareDiseases

To finish our #GriefAwarenessWeek takeover, we're looking to our creative designer's experience of grief as someone who lives with a #RareDisease. Joe, who lives with #Cystinosis, writes about losing his mum and how he now navigates the needs of his rare disease without the

Next up in our #GriefAwarenessWeek takeover, we're sharing an article by our editor, Nicola Miller. Here, Nicola writes about the uncomfortable aspects of opening up conversations around #Grief and how doing so can help set the journey ahead. Read at https://t.co/HdbrNqMM2Y

✨ It’s Day Three of our 24 Days of RARE Inspirations ✨ Introducing Oliver Mills, who lives with beta-mannosidosis and lysosomal Storage Disorder, and was nominated by his mother, Laurel. 💬 Here’s what Laurel had to say about Oliver: “In 2014, Oliver was diagnosed with

Grab a sneak peak of our interview with Doug Sproule, chief medical officer, ML solutions, a BridgeBio company, on FORTIFY, a phase 3 study for limb-girdle muscular dystrophy. Watch here: https://t.co/g6BSsLgP06

Let's continue with our #GriefAwarenessWeek takeover. Moving forward after a death has no rule book—remember, it's a marathon, not a sprint. Check out our Living with RARE #Grief resource to help understand these difficult feelings. Continue reading at https://t.co/f9hpra5585

Continuing our #GriefAwarenessWeek takeover, we're looking at Meaningful conversations, written by our Becky Pender. Here, Becky discusses the importance of the Hospice UK Dying Matters campaign. And how in our #RareDisease communities, the grief journey often starts long

Michael McGrath MBE, founder and CEO, Muscle Help Foundation, sets the scene on a substantial body of work driven by the organisation in partnership with the University of Hertfordshire, which looks at how we can better understand and support families through grief and

Today we're having a special takeover day to mark the start of #GriefAwareness Week—a topic that profoundly impacts many of us in #RareDisease communities. First, we're sharing our advice for navigating finance following a #Bereavement and ways that will help to make the whole

Huge thanks for joining our Tuesday Takeover with RARE Revolution Magazine! Want to learn more about CUL3? Visit https://t.co/LnwdqetwZX #CUL3 #CUL3SupportandAwareness #TuesdayTakeover

Families affected by CUL3-related neurodevelopmental disorder may be spread across continents, but the community is growing stronger every day. Connection matters, and visibility matters. When rare families find each other, the world feels a little less overwhelming and a lot

Behind every CUL3 diagnosis is a story - of resilience, learning, uncertainty, growth, and hope. We welcome you to read the stories of some of the individuals and families navigating a CUL3-related neurodevelopmental disorder diagnosis. https://t.co/de2I4vmGor #CUL3

Our blog is a growing hub for personal experiences, research updates, and practical resources related to CUL3-related neurodevelopmental disorder. Many topics also resonate across the wider rare genetic community. Take a look at what’s new: https://t.co/50cQHthdjs

Curious about the science behind CUL3? We maintain an up-to-date collection of published research articles to help families and professionals stay informed: https://t.co/v3bvI5DW3g We’re also part of the Simons Searchlight research program, a leading effort to better understand

✨ Welcome to Day Two of our 24 Days of RARE Inspirations ✨ Introducing our next nomination, Dana Pradunets, who lives with spinal muscular atrophy (SMA) type 1 and was nominated by her mother, Iryna. 💬 Here’s what Iryna had to say: "...I want to inform people so that they