National MPS Society

We are thrilled to share with you that REGENXBIO announced a strategic partnership with Nippon Shinyaku to develop and commercialize RGX-121 for the treatment of Mucopolysaccharidosis II (#MPSII), also known as Hunter syndrome, and RGX-111 for Mucopolysaccharidosis I (#MPSI), also known as Hurler syndrome. “Knowing [REGENXBIO] is keeping the focus on the commercialization and delivery of an approved product safely to our community illustrates their compassion and dedication to our community,” said National MPS Society President and CEO Terri Klein. “Congratulations!” Read more on their website: https://lnkd.in/eFgNHudW

Are you an adult with MPS IV (#MorquioSyndrome), or do you know someone who is? A study called "Sexual Health and MPS IV" at Boise State University is seeking volunteers. The goal of this study is to help people with MPS IV by raising awareness about an important topic that might be overlooked by doctors and healthcare teams: sexual health. Participants will take place in a virtual interview where they will share their experiences surrounding getting information on sexual health from a healthcare professional. The results will be used to help create a sexual health and relationship resource for people with MPS IV. Anyone 18 years of age or older with MPS IV can join the study, and the study is open to U.S. and international residents. If you're interested in participating, please email Katie Mazzon at [email protected]. #MPS #MPSIV

So pleased to see this news! For far too long, we at the National MPS Society have has so few options to convey for neuropathic MPSs like #Huntersyndrome (MPS II) and #Sanfilipposyndrome (MPS III) . I know that these communities will be so pleased to see this developement. Congratulations to Denali Therapeutics and the FDA, for pushing forward for our communities. #raredisease #mucopolysaccharidosis #huntersyndrome #sanfilipposyndrome #drugdevelopment https://lnkd.in/egpzQWpJ

📢 Exciting News for the MPS IIIA Community! Ultragenyx has announced plans to expand the Transpher A clinical trial for UX111 (formerly ABO-102) by introducing a fourth Cohort! This cohort aims to provide additional insights into the investigational gene therapy. Learn more at clinicaltrials.gov. The sites will begin engaging with caregivers on January 13, 2025. Please respect this timeline as they finalize preparations. Caregivers interested in learning more should consult their child’s physician or contact the trial sites starting on January 13. We are so grateful for Ultragenyx's commitment to families affected by Sanfilippo syndrome. Thank you for inspiring hope for a brighter future. #MPS #RareDisease #Sanfilippo #MPSIII

We had so much fun with you all over the last couple of days... Mark your calendars for when we do it all again next year at the 39th Annual Family & Scientific Conference in Denver, Colorado! 🎉🥳 #NationalMPSSociety #MPS #ML #RareDisease #HurlerSyndrome #HunterSyndrome #Sanfilippo #MorquioSyndrome #MaroteauxLamySyndrome #SlySyndrome #HyaluronidaseSyndrome

Time is running out— act now to protect critical legislation like the Creating Hope Reauthorization Act (H.R. 7384/S. 4583), Pediatric Priority Review Voucher (H.R. 5262/S. 1851), RARE Act (H.R. 7383/S. 1214), and more. It only takes two minutes to submit your statement on the National Organization for Rare Disorders, Inc. (NORD)'s website: https://ow.ly/8XVG50Uu3Lp #NationalMPSSociety #50Years #DisneyWorld #MPS #ML #RareDisease #HurlerSyndrome #HunterSyndrome #Sanfilippo #MorquioSyndrome #MaroteauxLamySyndrome #SlySyndrome #HyaluronidaseSyndrome

We’re excited to attend the National MPS Society’s Annual Family Conference this week in Orlando, FL, to honor and celebrate the organization’s 50 years of support, strength, and success for MPS families. REGENXBIO’s CEO, Curran Simpson, will join industry leaders for the CEO Fireside Chat and Dawn Phillips will share an update on our RGX-121 clinical program for the treatment of MPS II. We look forward to seeing the MPS community there!

The end of this year’s Congressional session is right around the corner! We need YOUR help advocating for several life-saving #RareDisease policy proposals. Urge your lawmaker to stand up for the rare disease community! ➡️ https://bit.ly/3VvveBy

🌟✨ Celebrate, Connect, and Discover: Join Us at the 38th Annual Family & Scientific Conference! ✨🌟 December 19-21, 2024, the National MPS Society is bringing the MPS and ML community together in Orlando, Florida, to celebrate 50 years of hope and progress! Our 38th Annual Family & Scientific Conference will feature cutting-edge research, meaningful connections, and opportunities to honor the incredible milestones we’ve achieved together. This year, we’re adding a touch of magic! While you engage in impactful sessions and connect with others, you'll also have the chance to make memories at nearby Walt Disney World. 💼 Whether you’re a researcher, family member, advocate, or supporter, this is your chance to make an impact and be inspired. 🌐 Register now: https://ow.ly/8Svn50UcZR5 ✨ Check out our free Disney tips: https://ow.ly/e0C850UcZOp Whether you’re coming for the science, the connections, or the celebrations, this is one conference you won’t want to miss. Let’s make magic together in Orlando! 🏰💜 #NMPSSC24 #NationalMPSSociety #50Years #DisneyWorld #MPS #ML #RareDisease #HurlerSyndrome #HunterSyndrome #Sanfilippo #MorquioSyndrome #MaroteauxLamySyndrome #SlySyndrome #HyaluronidaseSyndrome

Joan Eppehimer, aunt to board member Jason Madison, recently joined the Forrager podcast to discuss her popcorn business-- and, along the way, she spread the word about #MPS and the Society! Check it out: https://ow.ly/t3Pv50UaQ1L #MPS #ML #RareDisease #HurlerSyndrome #HunterSyndrome #Sanfilippo #MorquioSyndrome #MaroteauxLamySyndrome #SlySyndrome #HyaluronidaseSyndrome