Encouraging interim data has been shared from two studies of risdiplam (RG7916), a drug being researched as a potential treatment for spinal muscular atrophy types 1, 2, and 3. For…
According to a story from MedCity News, a recent study revealed a potential weakness of CAR-T cell immunotherapies that reveals the need for precision and caution in the extraction process.…
How it all started Joy Milne is a retired nurse who helped diagnose her husband with Parkinson's disease. 12 years before the official diagnosis, Joy noticed a change in her…
A past, current, and future problem – underrepresentation in clinical trials, particularly for rare diseases. Why does representation matter? Besides, you know, the basics of equality, drugs can react differently…
October is Dysautonomia Awareness Month! Before we get into advocacy, let's touch on basic awareness... What is dysautonomia? Dysautonomia is the umbrella term that encompasses several different conditions that cause a…
Lindsey Sutton showed signs of familial chylomicronemia syndrome as early as five weeks old. Sutton is now 28 years old. Because of her condition, she must adhere to a very…
A Texas company, which opened in 2015, promised a new treatment for a number of conditions. Simply coming in and subscribing to a routine of IV treatments could treat cancer,…
According to a story from wtvbam.com, the drug development company Alnylam Pharmaceuticals has recently stated that it is looking less likely that its gene silencing therapy givosiran will be getting…
Just days after my CVID diagnosis, I was invited to a support dinner. It was at this support dinner that I became aware of how unique my situation was; I…
According to a story from McGill University, a group of Canadian researchers have discovered a mutation in the French-Canadian population that is linked to diseases including multiple system atrophy, Parkinson's…
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