Category Archives: News
Shire has announced that they will be initiating a Natural History study for MPS IIIB
Official Title: “An Observational Prospective Natural History Study of Patients With Mucopolysaccharidosis Type III (MPS IIIB, Sanfilippo Syndrome Type B)” The purpose of this study is to evaluate the natural course …
New test spots early signs of inherited metabolic disorders
New test spots early signs of inherited metabolic disorders A team of scientists, led by researchers at the University of California, San Diego School of Medicine and Zacharon Pharmaceuticals, have …
Ultragenyx Pharmaceutical Inc. announced it has in-licensed an enzyme replacement therapy program to treat MPS VII
Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced it has in-licensed an enzyme replacement therapy program from St. Louis University …
Join us! National MPS Society endorses the Unlocking Lifesaving Treatments for Rare diseases Act of 2012
Help support ULTRA! Take action by contacting your legislators! In December 2011, U.S. Representatives Cliff Stearns (R-FL) and Ed Towns (D-NY), two senior members of the Energy and Commerce Committee, introduced …
Genzyme announces 2012 Co-Pay Assistance Program
Genzyme’s Co-Pay Assistance Program will help eligible individuals who are prescribed treatment with one of Genzyme’s enzyme replacement therapies with their eligible drug related out-of-pocket expenses, including co-pays, co-insurance and …
Shire announces 2012 OnePath Co-pay Assistance Program
Shire HGT is pleased to announce its new Co-pay Assistance Program for 2012 with 100% coverage for eligible expenses. Shire will assist eligible patients in the United States who have commercial insurance with …
Hunter Parents Community website
In October, the Hunter Parents Community, an online community sponsored by Shire, was launched. The website is an exclusive forum for primary caregivers of children with Hunter Syndrome to connect and share …
Graduate Study Recruitment Announcement
A PhD student at Wurzweiler School of Social Work at Yeshiva University is conducting a study of parents of children with rare disorders who had a delayed diagnosis. The study …
National MPS Society endorses the Lifespan Respite Reauthorization Act
On October 26, 2011, Representative Jim Langevin (D-RI) and Representative Catherine McMorris Rodgers (R-WA) introduced the Lifespan Respite Reauthorization Act (HR 3266). The Act reauthorizes the Lifespan Respite Program at a funding level of $50 …
3rd Annual EveryLife Art Contest
RareArtist.org and the EveryLife Foundation for Rare Diseases invite you to participate in the third annual EveryLife Art Contest! For contest rules and how to enter, please visit www.rareartist.org/main/contest_rule. PURPOSE: To …
