Thanks to all who attended our Virtual Family Conference on September 11-12th. It was a one-of-a-kind event, as we navigated new territory in a virtual environment. We hope you left with some informative and memorable takeaways from our Friday night breakout sessions, Saturday science and care management presentations, sponsor booths, and Camp Courage activities.
The webinar presentations from the conference are now available on-demand on YouTube!
If you weren’t able to attend live or would like to re-visit anything you missed, we invite you to click here to view our Family Conference YouTube playlist.
Thanks for making this a great event and we hope to see you all in-person for our 2021 Family Conference in Nashville!
Below is a list of summaries of the webinar presentations:
“COVID-19 updates for MPS Patients”
Dr. Heather Lau delivers an overview of the COVID-19 pandemic, some recent U.S. statistics, and the varying ways it can impact the MPS community. Read More
“MPS General Overview”
Dr. Joseph Muenzer presents a general overview of Mucopolysaccharidosis (MPS) and Mucolipidosis (ML). Read More
“Newborn Screening – What is It, How Will It Help the Diagnostic Journey”
Dr. Barbara Burton gives an overview of newborn screening, its current availability across the country, and the positive impact it can have on early detection of MPS and ML. Read More
“Natural History of MPS Disorders”
Dr. Elsa Shapiro delivers an overview of the effects of MPS on neurocognitive functions, how they differ in each syndrome type, and the effectiveness of transplant and enzyme replacement therapy for each. Read More
“Neurobehavior Manifestations and Other Considerations in MPS”
Dr.Julie Eisengart explores the likelihood that all MPS types have some underappreciated neurobehavioral symptoms, many of which could be the indirect effect of somatic symptoms. Read More
“Orthopedic Management of the Mucopolysaccharidoses”
Dr. Klane White provides a visual overview of pediatric orthopedics and the prevention and treatment of musculoskeletal disorders in children with MPS. Read More
“Airway Problems of Patients with Mucopolysaccharidosis”
Dr. Robert Wood discusses why individuals with MPS have a strong tendency to develop common airway problems, and the surgical and non-surgical recommendations to treat them. Read More
“Developing a Mucolipidosis Research Collaborative Network”
One year since the formation of the MCRN, the network continues to meet monthly, with the primary function to coordinate several labs focused on ML research. Read More
“Gene Therapy Updates and Pharmacological Presentations”
National MPS Society Chief Scientific Officer, Dr Matthew Ellinwood spoke on advances in a subset of gene therapy that involves gene editing, and an overview of novel treatment approaches involving drugs. Read More
“Advocacy and Newborn Screening”
The National MPS Society’s Advocacy Committee has monthly calls. There are over 25 members and they work in areas of newborn screening, appropriations language, legislative action alerts to members, COVID task force and partner with Everylife Foundation and the RDLA. Read More
“Mission Dollars, Family Fundraisers and the National Bake Sale”
The Fundraising presentation shared that in-person fundraising collapsed because of the pandemic this year. They worked hard to move towards virtual events and found success! Read More