National MPS Society

? Welcome Dr. Colleen Kraft to the National MPS Society! ? We are thrilled to welcome Dr. Colleen Kraft to our Board of Directors as she joins our mission to support families, advance research, and advocate for those affected by MPS and ML. Dr. Kraft is a Professor in the Department of Pathology and Laboratory Medicine, and the Department of Medicine, Division of Infectious Diseases, at Emory University. She is also a past President of the American Society for Microbiology. "I am elated to have Dr. Kraft join the Board," says Terri Klein, President & CEO at the National MPS Society. "We are so fortunate to have her, and her skills, here with us." You can learn more about Dr. Kraft by reading her bio on our website: https://ow.ly/a95H50VpCMi #MPS #ML #NationalMPSSociety #HurlerSyndrome #HunterSyndrome #Sanfilippo #MorquioSyndrome #MaroteauxLamySyndrome #SlySyndrome #HyaluronidaseSyndrome

Attention Advocates! Our March Advocacy meeting has been moved to Friday, March 28, at 1 PM EST. We hope you'll join us for an important discussion on state proclamations for MPS Awareness Day ?? Register now: https://ow.ly/s0W150Vokkm #MPSAwareness #HurlerSyndrome #HunterSyndrome #Sanfilippo #MorquioSyndrome #MaroteauxLamySyndrome #SlySyndrome #HyaluronidaseSyndrome

??Virginia Governor Glenn Youngkin approved HB 1782, a bill that mandates the state to consider national recommendations when determining which conditions newborns are screened for at birth. ??This ensures that newborns are screened for pediatric-onset conditions that are devastating but treatable when diagnosed early, providing opportunities for lifesaving medical interventions. The passage of this bill highlights the importance of bipartisan cooperation in advancing public health initiatives. To learn more visit: https://lnkd.in/e8eMstUk #newbornscreening #virginia #RUSP

Excited to share our latest paper from the Cherqui lab, demonstrating the beneficial effects of HPSC transplantation in our new MPS IIIC mouse model. Thank you to everyone who contributed to this project, and special thanks to the National MPS Society and Cure Sanfilippo Foundation for funding this work!!

It's our honor to introduce you to Stacy and Matthew (MPS I), whose mother, Nancy, recently shared their story with us. Did you know that Stacy was the first patient to receive an unrelated bone marrow transplant at the University of Iowa? Learn more about them on our website: https://ow.ly/WI0r50VjkmY #MPSAwareness #MPS #MPSI #HurlerSyndrome

REMINDER: Join us for a Virtual Science & Research Town Hall! ?? March 26 ? 7 PM EST Meet our new Chief Scientific Officer, Dr. Scott Loiler, and hear the latest on AAV gene therapy from him and Dr. Matthew Ellinwood, Director of Science and Research. Don’t miss this opportunity to learn and ask questions! ?? Register now: https://ow.ly/AHMs50VgM1O #RareDiseaseResearch #GeneTherapy #MPS #HurlerSyndrome #HunterSyndrome #Sanfilippo #MorquioSyndrome #MaroteauxLamySyndrome #SlySyndrome #HyaluronidaseSyndrome #MPSAwareness

Family planning is an important topic for many in our community. This month, our Pathways Support Group is diving into in vitro fertilization (IVF). Join facilitators Stephanie Cozine, Lisa Berry, and Charlotte Barrett-Weber for an insightful discussion covering IVF education, personal experiences, and a Q&A session. ?? March 18, 2025 ? 12 PM ET Register now: https://ow.ly/7qPm50Vf0ty #IVF #HurlerSyndrome #HunterSyndrome #Sanfilippo #MorquioSyndrome #MaroteauxLamySyndrome #SlySyndrome #HyaluronidaseSyndrome #MPSAwareness

? Save the Date! ? The National MPS Society’s Masquerade Gala is coming to Boston, MA, on October 11, 2025! ???? Join us for an unforgettable evening celebrating progress, hope, and the incredible MPS community. More details to come—stay tuned! Interested in sponsoring or learning more? Contact our Director of Development, Wendy Spaulding, at [email protected] or 919-806-0101!

We have incredible news for our MPS II (Hunter ) Community today. REGENXBIO has announced the filing of their rolling BLA for the first-ever AAV intracisternal Gene Therapy for MPS II. Addressing neuropathic disease is critical for our boys. We are elated, and answers are on the horizon. A decision for an approved therapy will come by the end of 2025. Thank you to everyone who participated and worked so hard on this clinical study. The National MPS Society will stay vigilant until everyone we support has treatment and someday, a cure. #connectedtogether https://lnkd.in/e46x6vuQ

So excited about this news! Congratulations REGENXBIO ??