National MPS Society

??? 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

?? The RDCRN Diversity Committee needs your help! They’re seeking individuals from rare disease communities—patients and caregivers—to participate in a study aimed at understanding barriers that prevent underrepresented racial and ethnic populations from joining rare disease clinical trials and research studies. ?? Details: A series of three focus groups will be held from December 11, 2024, through February 25, 2025. Learn more and get involved: https://ow.ly/ZtTJ50Uaef7 Please help spread the word by sharing the recruitment flyer across your networks— together, we can help make rare disease research more inclusive and representative. ?? #RareDiseases #DiversityInResearch #PatientVoices

Over the weekend, staff and Society members had the pleasure of attending the grand opening of the Muenzer MPS Research & Treatment Center in Chapel Hill, North Carolina. Dr. Muenzer has had a profound and lasting impact on the MPS community through his dedication to research, patient care, and advocacy. We are so grateful for his efforts, his commitment to our community, and for the wonderful, concrete reminder of his legacy that is the new treatment center #MPS #ML #RareDisease #HurlerSyndrome #HunterSyndrome #Sanfilippo #MorquioSyndrome #MaroteauxLamySyndrome #SlySyndrome #HyaluronidaseSyndrome

Since 2012, the FDA has awarded Priority Review Vouchers (PRVs) to companies developing life-saving and innovative therapies for children with rare diseases. This incentive has been instrumental in advancing treatments for conditions like MPS, ML, and related disorders. The Creating Hope Reauthorization Act (S. 4583) aims to extend the Pediatric Rare Disease Priority Review Voucher program through 2030. However, for this critical program to continue driving therapeutic breakthroughs, Congress must pass this legislation before the December 20, 2024 deadline. Help us advocate for the continuation of this legislation by sending a message directly to your Senators using this quick, easy tool from National Organization for Rare Disorders, Inc. (NORD): https://ow.ly/5n1u50TUBRO #MPS #ML #RareDisease #HurlerSyndrome #HunterSyndrome #Sanfilippo #MorquioSyndrome #MaroteauxLamySyndrome #SlySyndrome #HyaluronidaseSyndrome

The FDA CBER OTP’s next RegenMedEd webinar?is set to take place at noon this Wednesday, October 30th! The webinar, "Finding Your Support Team While Participating in a Clinical Trial," will feature a panel of patients, care partners, and advocates who will share their own experiences with support systems throughout the duration of a clinical trial, tips on navigating logistics, and more. Register now on the FDA's website: https://ow.ly/sGgY50TULtv

Will we see you in Orlando, FL, this December? ???? Registration is still open for the 38th Annual Family Conference: https://ow.ly/NE3450TOJR9 If you're an adult (18+) with MPS or ML, you're also invited to join us for a special SPIRIT Conference on December 21st: https://ow.ly/3EzR50TOJRa #MPS #ML #RareDisease #HurlerSyndrome #HunterSyndrome #Sanfilippo #MorquioSyndrome #MaroteauxLamySyndrome #SlySyndrome #HyaluronidaseSyndrome

I am attending #NORDsummit, and Joni L Rutter is presenting on unlocking biomedical research for innovations and breakthroughs for rare diseases at @NORD. NIH is blazing new paths as the largest funder of biomedical research, with patients as partners ensuring equity. NCATS also focuses on the common processes underlying rare diseases to develop more research faster and quicker while complementing individual research applications. These messages are crucial to hear. It gives patient communities the understanding that we are pooling resources to escalate research to eventual understanding and therapeutic development. Acceleration is an action word all Parents and Patients want to hear. National Center for Advancing Translational Sciences (NCATS) #raredisease #urgency National MPS Society

October is Health Literacy month! ?? Did you know that the Society has developed a library of resources dedicated to helping you understand #MPS and #ML? You can access it anytime on our website: https://ow.ly/wjjP50TOJ6g #MPS #ML #RareDisease #HurlerSyndrome #HunterSyndrome #Sanfilippo #MorquioSyndrome #MaroteauxLamySyndrome #SlySyndrome #HyaluronidaseSyndrome

Thank you to everyone who attended the 2024 Long Beach Race for Rare! It is always a good day when we can gather in support of our mission ??