Usher Syndrome Ireland

Wishing the USH community, family, friends, and our incredible supporters a safe and joyful Merry Christmas and Happy Holidays. We'll see you in 2025, ready to kick off another exciting year together! ✨ #UsherSyndrome #USH #MerryChristmas #HappyHolidays

Exciting Update on Ultevursen for Usher Syndrome Type 2a (Exon 13) Some of you may recall that ProQR's trial for USH2a (exon 13) was halted in August 2022 due to financial difficulties, leaving its future uncertain. However, in December 2023, Laboratoires Théa (Théa) announced they had acquired the rights to ultevursen, paving the way for its continued development. Today, we are delighted to share some promising news: Sepul Bio, an innovative unit of Théa, has resumed the development of ultevursen. The first clinical participant has now been dosed in the LUNA Phase 2b clinical study, marking a major milestone for this RNA therapy. Ultevursen holds great promise for helping to prevent further vision loss in individuals with retinitis pigmentosa (RP) or non-syndromic RP caused by variants in exon 13 of the Usher syndrome type 2a gene. The LUNA Phase 2b Study The LUNA clinical study, formally known as SB-421a-006, is a two-year, double-masked, randomised, sham-controlled trial. It will enrol 81 participants, including both adults and children (aged eight and older) with RP due to exon 13 variants in the USH2a gene. Looking Ahead A community update is expected in early 2025, offering further insights into the progress of ultevursen’s development. To ensure you don’t miss important updates like this, we encourage you to sign up for our newsletter and stay connected with the latest developments in the Usher syndrome community: https://lnkd.in/eZF4vSVQ You can read the latest press release from Laboratoires Théa here: https://lnkd.in/eFgm8JQR #UsherSyndrome #RetinitisPigmentosa #RP #USH2a #USHInResearch #Thea #SepulBio #RNA #ClinicalTrial #ultevursen

⭐ What a year ⭐We’re thrilled to share the highlights from 2024 in our #USHWrapped2024 special! Top Moment: USH Connect 2024 Event Our second-ever in-person event was a highlight of the year! This day brought together families, individuals, and professionals for a blend of informative and inspiring keynote talks, group activities, and meaningful connection. We’re also very proud of the growth from our first event— longer sessions, more attendees, and new opportunities to connect. A Big First: First Step Towards Ireland’s USH Patient Registry Thanks to a generous grant from the Hospital Saturday Fund, we’ve taken the first step toward creating Ireland’s first dedicated Usher syndrome registry. Demonstrating its need and impact could help in securing improved support and tailored services, and research that could make a difference in the lives of those living with Usher syndrome. Raising More Awareness: New HCP Awareness Packs Launched In 2024, we distributed USH Awareness Packs to healthcare providers nationwide, spreading understanding of Usher syndrome and dual-sensory loss. These resources will help to improve patient experiences. Strongest Connections: USH Connect Parents Peer Support Group This year, we launched our first peer-support series for USH parents through the Supporting Parents initiative, creating safe spaces to share advice and find strength in shared experiences. Our Greatest Achievement: Building a Thriving Community Every milestone we've achieved this year has been fueled by your support and belief in our mission. Together, we’re making a difference for the USH community in Ireland. Let’s Make 2025 Even Bigger! Thanks to your generosity, 2024 was a huge success. You can help make 2025 even brighter by supporting our Christmas Fundraiser—every donation drives real change. Donate here: https://lnkd.in/eBM7U4zr #UsherSyndrome #UsherSyndromeIreland #RareDisease #RareDiseaseIreland #SupportUSH #USHResearch #USHFam #USHFamily #USHKids #UsherKids #Ushers #Christmas #Fundraiser #Wrapped2024 #SupportingParents #LearnAboutUSH #USHConnect

⭐ What a year ⭐We’re thrilled to share the highlights from 2024 in our #USHWrapped2024 special! Top Moment: USH Connect 2024 Event Our second-ever in-person event was a highlight of the year! This day brought together families, individuals, and professionals for a blend of informative and inspiring keynote talks, group activities, and meaningful connection. We’re also very proud of the growth from our first event— longer sessions, more attendees, and new opportunities to connect. A Big First: First Step Towards Ireland’s USH Patient Registry Thanks to a generous grant from the Hospital Saturday Fund, we’ve taken the first step toward creating Ireland’s first dedicated Usher syndrome registry. Demonstrating its need and impact could help in securing improved support and tailored services, and research that could make a difference in the lives of those living with Usher syndrome. Raising More Awareness: New HCP Awareness Packs Launched In 2024, we distributed USH Awareness Packs to healthcare providers nationwide, spreading understanding of Usher syndrome and dual-sensory loss. These resources will help to improve patient experiences. Strongest Connections: USH Connect Parents Peer Support Group This year, we launched our first peer-support series for USH parents through the Supporting Parents initiative, creating safe spaces to share advice and find strength in shared experiences. Our Greatest Achievement: Building a Thriving Community Every milestone we've achieved this year has been fueled by your support and belief in our mission. Together, we’re making a difference for the USH community in Ireland. Let’s Make 2025 Even Bigger! Thanks to your generosity, 2024 was a huge success. You can help make 2025 even brighter by supporting our Christmas Fundraiser—every donation drives real change. Donate here: https://lnkd.in/eBM7U4zr #UsherSyndrome #UsherSyndromeIreland #RareDisease #RareDiseaseIreland #SupportUSH #USHResearch #USHFam #USHFamily #USHKids #UsherKids #Ushers #Christmas #Fundraiser #Wrapped2024 #SupportingParents #LearnAboutUSH #USHConnect

❗IMPORTANT REMINDER We need YOUR opinion to guide important research! Usher Syndrome Ireland has partnered with Connect Research Ltd. to carry out a scoping project assessing the need for, and design of, a patient registry for the USH community in Ireland. This is a vital first step toward creating a registry that could shape future research and improve support services to truly meet the needs of those living with Usher syndrome. If you're over 18, please take a few minutes to complete this urgent survey today. Your feedback will help decide not only whether a registry is needed, but also what features it should include to best serve the USH community. Survey link: https://lnkd.in/evfixSjX Help us spread the word! Sharing this survey with others could make a huge difference in gathering diverse voices from across the USH community. #UsherSyndrome #Survey #UsherSyndromeIreland #Research #PatientRegistry #USH #UsherKids #USHFam #RareDisease ID: A woman is holding a sign that says 'Your Voice Matters'. There is also a reminder icon in the image. 

🌟 It's Charity Trustees Week! 🌟 We want to send a MASSIVE thank you to our incredible seven trustees! Your unwavering dedication, hard work, and countless sacrifices are the backbone of Usher Syndrome Ireland. Because of you, we continue to make a meaningful difference for everyone affected by Usher syndrome and their families. Your commitment drives our mission forward, creating brighter futures and offering much-needed support to our community. 👏 #TrusteesWeekIrl #ThankYou #ThankYouTrustees #UsherSyndrome #UsherSyndromeIreland #CommunityHeroes #NonProfit #MakingADifference #Volunteers #CharityTrusteesWeek #TogetherWeCan #RareDisease #RareDiseaseIreland Image Descriptions: 1. Six trustees - Trudy Matthews, Deborah Heffernan, Eugene Lynch, Carol Brill, Deirdre Leech, and Anne Shields - are standing together with smiles. 2. Trustee David Ashley poses with our safety officer Theresa, both smiling through an Instagram cutout frame.

🎅 Pull On a Jumper, Make a Difference! This year, we need your help more than ever. It’s been a tough time for fundraising, but with your support, we can bring some festive fun and hope to those affected by Usher Syndrome. On December 12th, join us for Christmas Jumper Day and help raise vital funds for Usher Syndrome Ireland. By taking part, you’re not just wearing a Christmas jumper, you’re helping to fund vital research, provide essential resources, and support families living with this rare condition. Even the smallest effort can make a real difference! Two Ways to Get Involved: 1. Wear & Donate: Put on your favourite Christmas jumper, make a donation to Usher Syndrome Ireland, and tag a friend to keep the festive chain going! 2. Host a Group Event: Get your classmates or colleagues to wear their most festive jumpers and ask everyone to chip in a small donation. It’s an easy and fun way to come together for a great cause. You can easily set up your fundraising page via iDonate.ie to collect donations from friends, family, and colleagues. There’s also a one-off donation button available on our iDonate page if you prefer to give a single contribution. Link to our iDonate page to set up a fundraiser/donate: https://lnkd.in/ekAy_RNA Share Your Festive Photos: Snap a picture, post it with #JumpersForUsher, and tag us to spread the word! Every jumper worn and every donation made helps us continue our vital work. #ChristmasJumperDay #UsherSyndrome #RareDisease #ChristmasJumper #Christmas #NonProfit #Fundraise #USH #Charity #SupportUSH

November updates coming shortly for our subscribers. Not signed up? Sign up here: https://lnkd.in/eWGH2Fc9 #UsherSyndrome #Updates #USHNews #USH #Research #MailingList

Here’s a sneak peek of our new animation for healthcare professionals, giving a quick overview of Usher syndrome - a rare genetic condition affecting both vision and hearing. Watch the full video on our YouTube channel here: https://lnkd.in/ez2zMHeP Don't forget to share to spread awareness! #UsherSyndrome #RareDisease #Healthcare #HCPs #RareIs #Healthcare #LearnAboutUSH #HealthcareProviders #HealthcareProfessionals #Awareness #USHAwareness  @Amgen @RareIs Video description: Animation opens with two healthcare workers, one of whom begins narrating, accompanied by on-screen text. The video then cuts to a still with the message: 'Watch the full video on our YouTube channel @ UsherSyndromeIreland.'

Your Voice Matters: Help Shape the Future of Usher Syndrome in Ireland We need YOUR voice to guide important research! Usher Syndrome Ireland has enlisted Connect Research Ltd. to conduct a scoping project to assess the need and design of a patient registry for the Usher community in Ireland. This is a crucial first step toward creating a registry that could shape future research and support services to better meet the real needs of those living with Usher syndrome. If you're over 18, please take a few minutes to complete this urgent survey today. Your feedback will help determine not only whether a registry is needed, but also what it should include to truly benefit the community. Survey link: https://lnkd.in/evfixSjX #UsherSyndrome #Survey #UsherSyndromeIreland #Research #PatientRegistry ID: A woman is holding a sign that says 'Your Voice Matters'.