Hattiesburg, Miss. – A teenager in Mississippi is living with a disease so rare that less than 20 people worldwide have ever been diagnosed with it. Symptoms for 16-year-old Trey Garczynski started when he was 11 years old. “I was just walking around, but just a few minutes later, my...
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HAMPTON, Conn. — A parent’s worst nightmare is that their child will suffer from a life-threatening disease with no cure on the horizon. But a family in Hampton said it’s putting all of its hopes into an experimental stem cell treatment and is traveling to Germany for the surgery. In...
The FDA and the Centers for Disease Control and Prevention (CDC) have launched an investigation into a potential connection between the Gardasil vaccine for the human papillomavirus (HPV) and a rare degenerative muscle disease. Concern over a connection between Gardasil and the rare disease — known as amyotrophic lateral sclerosis...
A teenager from North Somerset with a life-threatening illness is meeting the Prime Minister to ask him for more help in funding research into his condition. Matthew Barrett, 13, suffers from Duchenne Muscular Dystrophy – a muscle-wasting disease in men which cuts life expectancy to about 20 to 25 years....
FREDERICK, Md. and MINNEAPOLIS, Minn. — TeleRare Health™ launched a partnership today with a leading community health system in Maryland, with a focus on expanding patient access to genetic testing, diagnosis, and knowledgeable care for rare and genetics-based diseases. In the United States, a disease is considered rare if it...
SOUTH SAN FRANCISCO, Calif. – Tenaya Therapeutics, a biotechnology company with a mission to discover, develop and deliver curative therapies that address the underlying causes of heart disease, today announced the hires of Leone Patterson, MBA, as Chief Financial and Business Officer and Matthew Pollman, MD, MS, as Senior Vice...
GRAND RAPIDS, Mich. – Tetra Therapeutics, a clinical stage biopharmaceutical company focused on the development of compounds for the treatment of brain disorders associated with cognitive and memory deficits, notably BPN14770, a selective inhibitor of phosphodiesterase‐4D (PDE4D), announced changes to its executive team as the company marks one year since...
THE WOODLANDS, Texas — Ollie Rasberry has Metachromatic leukodystrophy (MLD), a rare and fatal disease. His big sister, Addi, has it, too. She is 5 years old, and the disease has already progressed rapidly in her. Life expectancy for a child living with their form of MLD is between 5...
LOS ANGELES – 54 honorees representing 23 states participated in the 2022 Donate Life Rose Parade® float, “Courage to Hope.” With ages ranging from 16 to 71 years old for riders and walkers, and from 9 days old, to 55 years old for floragraph honorees, these participants and their families...
BRONX NY — Neurologists often diagnose Alzheimer’s disease after evaluating patients during lengthy, in-person office visits. This poses a significant challenge for many groups, particularly people with limited access to specialized care, including people from historically marginalized groups and people living in rural areas. Albert Einstein College of Medicine has...