…expert in image acquisition and other methods of image analysis. Thus far, excellent manual tracing reliabilities on MPS I patients and preliminary data on brain volumes in 18 MPS I patients and 9 MPS II patients have been obtained. The results of the reliability study were presented at the WORLD symposium in February; this presentation is now being written for publication. It is attached to this progress report as well as the volumetric comparisons of attenuated MPS I and MPS II. In addition, clinical ratings scales (such as the Matheus…
…initiation of melatonin provides assessment of potential benefits. Melatonin is available in the following forms, tablet, capsule, liquid, lozenges (dissolves in the mouth), and sublingual tablets (dissolves under the tongue). The suggested dose of melatonin is 1mg – 10mg, and it’s recommended that the lowest dose be initially given. For optimal benefit, develop a bedtime routine that is followed every night and give the melatonin at the same time every night. Avoid use of lights in the bedroom, keeping the room as dark as possible. Melatonin may cease to work…
Shop the RARE Aware Store For logo items, we invite you to visit the RARE Aware Shop, created and managed by the Adult Resource Committee. Proceeds from these sales support adults diagnosed with MPS or ML. Shop Now 1 Our Mission The National MPS Society exists to cure, support and advocate for MPS and ML….
…for MPS II, Michael and I traveled up to Duke University for him to have a unrelated cord blood bone marrow transplant on December 26, 2008. This has been a trying and difficult path being a single parent, but I would do it all over again if I had to in order to do whatever I could for my child. We would greatly appreciate your contributions with hope it can help find a cure for Michael and others affected by MPS. 1500 150 MPS II 19484 19485 19486 19487 19488…
…simply click the “Donate” button in the upper right to donate. Make sure to bookmark this page and come back often to check on my progress! 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. 2000 150 MPS IIIC 19412 19413 19414 19415 19416…
…company is enrolling patients in a clinical trial for the drug at two sites. Results from the Phase IIa study are expected during the first semester of 2019. For the most up-to-date information on odiparcil, click here. JCR Pharmaceuticals JCR Pharmaceuticals utilizes “J-Brain Cargo®” technology to deliver active drug molecules to the central nervous system through the blood-brain barrier. JR-141 is recruiting for patients with MPS II in a phase III clinical trial. JR-171 is recruiting for patients with MPS I in a phase I/II clinical trial. Lysogene Lysogene is…
…was granted in 1986 shortly after the name of the newsletter was changed to Courage. 2000 The MPS Society 5k walk/runs began with seven events bringing in $100,000. April 2003 The first enzyme replacement therapy (ERT) for an MPS disease was approved by the FDA, Aldurazyme for MPS I. 1997 to 2004 Legislative advocacy efforts increased research funding from NIH for MPS and related diseases. Funding increased from $5.9 million in 1997 to $9.3 million in 2004. 2005 & 2006 Naglazyme, the ERT for MPS VI was approved in 2005,…
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…
…many countries in Europe. Elaprase was developed and is produced by Shire Pharmaceuticals and is given as a weekly infusion. For more information, visit www.shire.com or contact OnePathSM at 866-888-0660. OnePath provides assistance with insurance, product access, treatment centers and education about Elaprase and MPS II. MPS IVA Vimizim™ (elosulfase alfa), developed by BioMarin Pharmaceutical Inc. is the enzyme replacement therapy for individuals with MPS IVA (Morquio A syndrome.) Vimizim is administered weekly via intravenous infusion. BioMarin will offer support to patients through its BioMarin Patient & Physician Support (BPPS)…
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…
