Moving into a New Life with Duchenne

I recently started my internship at The Washington Post in Washington, D.C. Getting to this point was a culmination of blood, sweat, and tears throughout all levels of education, and it’s not going to get any easier in the professional world. Establishing myself…

It’s been 15 years since Sailormen, a Popeyes Louisiana Kitchen franchise, first partnered with the Muscular Dystrophy Association (MDA) to enhance the lives of patients. It’s keeping at it, netting $681,015 in its latest efforts. The money was raised at the 15th Annual Sailormen MDA Golf Classic and the…

Europe’s umbrella organization for 800 rare disease associations has developed a sweeping initiative to help the continent’s 30 million rare disease patients and their caregivers learn about their conditions, find assistance and receive treatment. Eurordis-Rare Diseases Europe hopes to improve the current piecemeal treatment and support program with a holistic,…

Biophytis SA is collaborating with AFM-Telethon to advance an oral pediatric formulation of Sarconeos (BIO101) for treating Duchenne muscular dystrophy (DMD). The pact calls for the French muscular dystrophy organization AFM-Telethon to give €400,000 (about $452,000) to France-based Biophytis  for further preclinical study. The funds also will go…

This column is a particularly nice one for me to write because of my love of bicycle riding. I began to bike in 1988. I was 29 years old and looking for a better way to stay active while enjoying the months of nice weather in Chicago. Stationary bicycles are…

To help make cutting-edge genetic research more accessible, and raise awareness of rare diseases such as Duchenne muscular dystrophy (DMD), YourDNA and Cure Rare Disease (CRD) are joining forces. The initiative hopes to increase awareness about the prevalence of rare disorders in the U.S. — and about treatment…

My early years in this world were spent being a girlie girl. Along with my stash of crowns and fake pearls were things like brightly colored nail polish and some mini lipsticks bought at Sally’s Beauty. Little did I know that as I grew older and my mitochondrial myopathy…

Watching YouTube videos allows me to simultaneously learn something new, laugh out loud, and connect with the gaming community. I never thought I could start a YouTube channel on my own. With the small number of videos I’ve produced on the…

People with rare diseases know that the right government policies can make a big difference in the quality of their own lives, and those of their caregivers. But most lawmakers aren’t experts in even one well-known disease — let alone the world’s estimated 7,000 rare disorders. So how does the…

Initially approved in the United States for Duchenne muscular dystrophy (DMD) patients age 5 and older, the corticosteroid Emflaza is now available for those as young as 2. The U.S. Food and Drug Administration (FDA) recently approved PTC Therapeutics’ supplemental New Drug Application (sNDA) for the…