Before my 2-year-old son, Alfie, was born, I was a primary school teacher. While I still work occasionally on a casual basis, it’s a much smaller part of my life than it once was. One of my favorite things about being a teacher was the constant learning — and…
Vita Therapeutics announced that it has raised $32 million in financing to advance its work into cell-based therapies for muscular dystrophy. “At Vita Therapeutics our mission is to deliver long-term disease-modifying cell engineered treatments for patients living with muscular dystrophies and other high unmet medical needs,” Douglas Falk, the…
What does it mean to live? Different people and places may prompt varied answers to this question. For example, in the United States, a person on artificial life-support is considered to be alive, while other people or cultures may not agree with this definition. So, what does it mean to…
CureDuchenne has launched the first comprehensive data hub to connect biosamples and clinical and genetic data from people with Duchenne and Becker muscular dystrophies — as well as disease carriers — with clinicians, researchers, and therapy developers. Called CureDuchenne Link, this next-generation data-sharing platform is expected…
To help patient advocacy leaders and their partners better understand how global health statistics codes — known as ICD codes — are assigned, updated, and revised in the U.S. health information system, the EveryLife Foundation for Rare Diseases is presenting a first-of-its-kind resource guide. The foundation created the…
My life has been quite busy lately. Between starting my full-time job at Bionews, helping produce two Call of Duty League YouTube shows, working on other freelance journalism projects, and managing my health, which gets progressively worse because of Duchenne muscular dystrophy, I’ve become a bit overwhelmed. When…
Those who wish to gain practical tools for living optimally with rare diseases are encouraged to attend the annual Living Rare Living Stronger Patient and Family Forum, hosted by the National Organization for Rare Disorders (NORD) and set this year for June 26-27. The conference brings together patients,…
The 15th Annual Jared Sweet Golf Outing, a Michigan event that benefits Parent Project Muscular Dystrophy (PPMD) and raises needed funds for research, is set for Saturday, June 19. To date, the outing has raised more than $160,000 for the PPMD. Since the event’s inception, the Sweet family,…
An analysis of microRNA molecules isolated from the blood of people with Duchenne muscular dystrophy (DMD) found alterations related to cholesterol metabolism, a study demonstrated. These findings suggest that the cholesterol metabolic pathway may be a potential therapeutic target for this most common type of muscular dystrophy, the…
Last June, my 2-year-old son, Alfie, had his first surgery to place a feeding tube. It has now been a year since the percutaneous endoscopic gastrostomy (PEG) tube replaced the nasogastric (NG) tube that previously passed down his nostril and throat. Alfie first had an NG tube for…
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