Pardon Our Dust: RAREDaily Under Construction

Thank you for visiting! Please continue to submit your patient stories to be featured in the launch of the new site Fall 2017! We appreciate your patience, please feel free to look through our archived articles.  With Hope, The Global Genes Team

Rare Disease Report: Is Sjorgen Syndrome a Rare Disease?

By James Radke at Rare Disease Report According to the Genetic and Rare Diseases Information Center (GARD) at the National Institutes of Health (NIH), Sjogren’s syndrome is not a rare disease, but based on data presented at the Annual European Congress of Rheumatology (EULAR 2017),…Continue Reading

CARES Foundation Works to Support the Congenital Adrenal Hyperplasia Community

(SOURCE) While the CARES Foundation is completely unknown to many people, there is a small but significant group of the population that heavily relies on the organization. That group is those affected by CAH — Congenital Adrenal Hyperplasia — a family of rare genetic diseases that…Continue Reading

Notre Dame Football Player Support Rare Disease Community Through Bowling Event

The Notre Dame football team traded in their football cleats for bowling shoes Sunday at the Strike & Spares Event Center in Mishawaka. The Notre Dame Chapter of Uplifting Athletes held its annual bowling event for the fourth consecutive year. This event is held to support the…Continue Reading

Rare Patient Starts Card Project for Hospitalized Kids

SOURCE Jen Rubino knows how it feels to be sick and in the hospital at a young age. The 22-year-old was diagnosed with a rare childhood bone disease at 10 years old. She has spent years in and out of hospitals and has had more…Continue Reading

New Data Show Survival Improvement with Adcetris in Hodgkin Lymphoma Patients

by Mathew Shanley for Rare Disease Report Earlier today, Takeda and Seattle Genetics announced promising results from its Phase 3 ECHELON-1 Clinical Trial that evaluated Adcetris (brentuximab vedotin) in Frontline Advanced Hodgkin Lymphoma. The randomized trial met its primary endpoint, demonstrating a statistically significant improvement in…Continue Reading

Doctor, CSO, and Father: Hunting for Answers for Fragile X Children

Dr. Michael Tranfaglia serves as Medical Director and Chief Scientific Officer of FRAXA, a nonprofit organization based in Newburyport, Massachusetts, which is committed to finding a cure for fragile X syndrome. Dr. Tranfaglia is responsible for coordinating the FRAXA foundation’s research strategy and works with…Continue Reading

CheckRare: FDA Approves Haegarda to Treat HAE

The U.S. Food and Drug Administration today approved Haegarda, the first C1 Esterase Inhibitor (Human) for subcutaneous administration to prevent Hereditary Angioedema (HAE) attacks in adolescent and adult patients. The subcutaneous route of administration allows for easier at-home self-injection by the patient or caregiver, once…Continue Reading

Patient-Inspired Research Uncovers New Link to Rare Disorder

Meeting a young patient with Zellweger syndrome, a rare, life-threatening genetic disease, started a scientific investigation that culminated with an unexpected discovery. The condition, also known as peroxisomal biogenesis disorder, had been linked only to lipid or fat metabolism. Now, as a team of scientists…Continue Reading

“Pain Won’t Stop Me From Making Memories,” CRPS Patient Takes Back Up Her Favorite Winter Sport

by Kerry Hussey I’m 30 years old now, but was diagnosed with RSD/CRPS back when I was 13-years-old. I was one of the “lucky” ones, where it only took three months to put a name to what I was going through, but that didn’t make…Continue Reading

NPR: A Couple’s Quest To Stop A Rare Disease Before It Takes One Of Them

by Rae Ellen Bichell In 2010, Sonia Vallabh watched her mom, Kamni Vallabh, die in a really horrible way. First, her mom’s memory started to go, then she lost the ability to reason. Sonia says it was like watching someone get unplugged from the world. By the…Continue Reading