WATKINS GLEN, N.Y. (WETM) – Liam Hickey, 6, is one in a million, so it is only fitting he had a special experience at Watkins Glen International race track. The Hickey family has been through some challenging times. Liam was diagnosed with a rare skeletal disease and has undergone several...
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COLUMBUS, Ohio — An Ohio mom says it took her months to say out loud that her son had a rare disease. “As soon as I Googled it, I knew what we were facing,” said Kelly Maynard. “That kicked off the worst six months of my life.” Her son, Jackson,...
WHITE PLAINS, N.Y. – Oligomerix, Inc., a privately held company pioneering the development of small molecule therapeutics targeting tau for rare neurodegenerative and Alzheimer’s diseases, today announced the appointment of William Erhardt, M.D., Chief Medical Officer of Oligomerix, to its Board of Directors. “As Chief Medical Officer, Bill has provided...
On a Wednesday morning in March 2021, Louise Storey, a champion rower who represented New Zealand at the London Olympics and several World Championships, pulled into a car park next to a Cambridge petrol station for a phone call that would change her family’s life. Louise (née Trappitt) had dropped...
RemeGen Co., Ltd., a commercial-stage biotechnology company, continues to strengthen its social responsibility and rare disease advocacy in unison with Rare Disease Day 2023's message of continuing to increase visibility and generate change for the millions of people globally living with a rare disease, their families and carers
WASHINGTON, D.C. —- Tuesday, U.S. Sen. Ben Ray Luján (D-N.M.), a member of the Senate Committee on Health, Education, Labor and Pensions, and U.S. Sen. Martin Heinrich (D-N.M.) reintroduced the Cerebral Cavernous Malformations Clinical Awareness, Research and Education Act (CCM-CARE) which would expand National Institutes of Health (NIH) research related...
Hattie Foster looks up to the sky. She’s 6 years old. The sun is shining, and she’s buzzing with energy. “Where’s the moon?” she asks whomever is listening. Her older brother, 9-year-old Charlie, climbs across the nearby jungle gym at a Prairie Village playground as her younger brother, 3-year-old Will,...
BOSTON and LONDON – Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the appointment of two gene therapy and industry experts, Fulvio Mavilio, Ph.D., and Nicoletta Loggia, Ph.D., to serve as chief scientific officer and chief technical officer, respectively. The company also announced the promotion of Leslie Meltzer, Ph.D.,...
QUINCY, Mass. – The Orphan Drug Act (ODA) celebrates its birthday on January 3. The year 2024 marks 41 years of revolutionizing drug development for rare diseases and helping millions of patients in the process. More than 25 million Americans have some kind of rare disease, defined as a condition...
At the end of 2011, I experienced an acute episode of diarrhea and vomiting for hours. I had returned home from New York City, consuming pasta with bacon bits and drinking a few glasses of red wine. By morning, I seemed better and went about my day caring for family, failing to visit a...