…support that has helped us progress this drug development from an idea to an NIH grant supported program and recently to a partnership with Pfizer. The following is a brief description of our progress over the last year: Testing the In Vivo Efficacy of Lead Compounds. Through the first year of support, we identified a series of analogs with improved potency in cellular models of MPS. Our most potent analogs are active in the 100 to 500 nM range, a 100-fold improvement from the original compound. Over the last year,…
…initial focus on MPS II, we are launching the 100 Patient Project to harness the revolutionary insights that can come from Whole Genome Sequencing (WGS) to better understand how genetic variation can inform future improvements to MPS disease management. Sign up to join us today! We invite those with MPS II or their caregivers to join the Luna portal created for the 100 Patient Project and the Unlock MPS Registry. You will fill out two introductory surveys, one with very basic information and a second Registry Intake Survey with details…
…treated or not treated with cochlear implant (102.10 or 102.11 for children) Applicants with MPS or ML also are eligible for an expedited application process called compassionate allowances, which seeks to pay benefits to those with very severe and obvious disabilities. Compassionate allowances enable individuals to qualify based on minimal medical evidence. The Application Process The application can be completed online or with a representative from the SSA. Applications for children must be completed in person and can’t be submitted online, however an adult can choose either method. http://www.disability-benefits-help.org/content/application-process Before…
…NBS! Every fourth Wednesday of the Month at 1:00pm EST: National MPS Society’s Advocacy Committee Meeting – Email info@mpssociety.org to receive the Zoom login information for this monthly meeting! May 15: Speakers’ Bureau Virtual Capitol Hill Meetings – Advocates from across the country will meet with law and policy-makers in Washington D.C. Napa Race for a Cure Sunday, April 28th 8:30 – 12:00 EDT Register Online Keller’s 5k for MPS Saturday, May 04th 7:00 – 12:00 EDT Register Online Million Dollar Bike Ride Saturday, June 08th Register Online Long…
Link to purchase disposable swim diapers from My Pool Pal www.mypoolpal.com Link to Duraline Medical Products for specialty undergarments www.arktherapeutic.com Link to Adaptive Mall.com, a resource for medical equipment southpawenterprises.com Vehicle Conversion…
…Europe for long-term enzyme replacement therapy in patients with a confirmed diagnosis of MPS I, to treat the non-neurological manifestations of the disease. Aldurazyme was developed by BioMarin and SanofiGenzyme under a joint venture agreement that assigns commercial manufacturing responsibilities to BioMarin, and worldwide sales and marketing responsibilities to SanofiGenzyme. Visit www.aldurazyme.com or contact SanofiGenzyme at 800-745-4447 for more information. MPS II Elaprase™ (idursulfase) is a long-term enzyme replacement therapy for patients with a confirmed diagnosis of MPS II and has been approved for use in the U.S., Canada and…
Center for Medicare and Medicaid Services https://www.cms.gov Social Security Online https://www.ssa.gov United States Department of Health and Human Services https://www.hhs.gov National Disabilities Council https://www.ncd.gov…
…back often to check on our progress! Michelle’s journey from diagnosis at 1 year to her bone marrow transplant at 20 months old to living with MPS1 are shared at www.caringbridge.com/visit/michellehopkins The mission of the National MPS Society is to fund research and provide support for families affected by MPS and related diseases. Please help our family and support the National MPS Society. Every donation will make a difference in the lives of those affected by MPS and related diseases. Please visit www.mpssociety.org for more information. 10000 400 MPS I…
…care at home. FCA now offers programs at national, state and local levels to support and sustain caregivers. www.caregiver.org National Family Caregivers Association The National Family Caregivers Association (NFCA) supports, empowers, educates, and speaks up for the more than 50 million Americans who care for a chronically ill, aged, or disabled loved one. NFCA reaches across the boundaries of different diagnoses, different relationships and different life stages to address the common needs and concerns of all family caregivers. www.nfcacares.org The Courageous Parents Network CPN is a destination created by parents,…
…in Cooper’s life, and we are certain his quality of life is benefitting from treatment. We are lucky that the research (and the funding it required) to find a treatment for Morquio was completed by the time we were diagnosed. But we are not done. Funding research specific to MPS diseases is critical to the next step – finding a CURE. 100% of your donation will fund life-saving research to find a cure for our little boy and others like him. About Cooper: Cooper is a sports nut. He loves…
