More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine was approved under emergency use authorization instead of the routine process, the EveryLife Foundation for Rare Disease reports. These findings…
A chaperone protein called DnaJB8 has an internal molecular “switch” that likely regulates how cells dispose of protein aggregates, like those that cause Huntington’s disease. These findings were detailed in the study “Regulatory inter-domain interactions influence Hsp70 recruitment to the DnaJB8 chaperone,” published in Nature Communications. Huntington’s is…
One of the characteristics of a good journalist is being able to digest a lot of information, such as the entirety of a study, and then concisely present the most pertinent and interesting facts in a 700-word story. I have written many of these articles in my career, and I…
The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a second year, the #RAREis Scholarship Fund — supported by Horizon Therapeutics – will award 35 one-time scholarships, each…
A new international consortium, Stem Cells for Huntington’s Disease (SC4HD), has been formed to help facilitate the development of stem cell-based therapies to treat the disorder. The consortium was presented in the Journal of Huntington’s Disease, in the paper, “Stem Cells for Huntington’s Disease (SC4HD): An International…
Over Easter weekend, my mother-in-law, Edwina, visited. My wife, Jill, and I are always happy to see her, and as usual, the conversation turned to the latest news in the family. One notable occurrence was a situation involving her brother’s mother-in-law, Lucille, whose health at 97 years old is fragile.
Having a parent with Huntington’s disease influences my life in many ways, most of which I’m likely unaware of. It influences everything from my plans for the future to how I view the world. My mom raised me to be independent (like that…
Anima Biotech has partnered with Takeda Pharmaceuticals to develop small molecules that control mRNA translation — the process by which the information encoded in a person’s DNA is “translated” into a protein — as potential therapies for neurological disorders with a genetic cause, including Huntington’s disease. Huntington’s…
“Do you believe in miracles?” Sports announcer Al Michaels uttered that sentence on national television in February 1980 during an Olympic hockey game between the United States and the Soviet Union. During the game, which was later dubbed the “Miracle on Ice,” the Americans were heavy underdogs. But that…
As anyone affected by a rare disease knows, treating the illness while trying to go about everyday life is an expensive undertaking. But exactly how expensive — in terms of direct and indirect costs across rare disease populations — might still come as a surprise: almost…
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