Rare Disease 101 Rare Disease Day is an international awareness campaign that takes place on the last day of February each year. It is the one day the world comes together to recognize people with rare diseases with the goal of raising awareness among the general public and decision-makers about rare diseases. The global theme for Rare Disease Day 2018 is patients and research. For patients, their families and caregivers, getting up to speed on […]
By Ann Allen “Find a group of people who challenge and inspire you, spend a lot of time with them, and it will change your life.” ~Amy Poehler Ah, you made it through the holidays. Now what? Did you make a New Year’s resolution? Did it sound something like mine and contain words like healthy eating, exercise, less stress? I know. We try. We’re busy. Our people come first. I am not a personal trainer, motivational […]
Mepsevii (Vestronidase Alfa) is the First Therapy for Progressive and Debilitating Rare Genetic Disease Mucopolysaccharidosis Vii Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, recently announced that the U.S. Food and Drug Administration (FDA) has approved MEPSEVII™ (vestronidase alfa), the first medicine approved for the treatment of children and adults with Mucopolysaccharidosis VII (MPS VII, Sly syndrome). MEPSEVII is an enzyme replacement therapy designed to […]
by Ann Allen ~The everyday kindness of the back roads, more than makes up for the agony of the headlines By Charles Kuralt Life is beautiful. Ah, such a cliché, but sometimes it feels like that when people are put in your life at the right time, in the right place, and wearing running shoes. This is the story of how my family joined the running community. On Sunday, April 14, 2013, a woman approached […]
To study the impact of rare disease on unpaid friends and family members who provide care (known as “family caregivers”), the National Alliance for Caregiving and Global Genes, are launching a “first-of-its-kind” national snapshot of rare disease caregivers. This fall, the two groups plan to release a national survey aimed at collecting feedback from over 1,000 family caregivers of children and adults with rare diseases. The survey will be open from Wednesday, September 13 through […]
