Currently, 95% of rare diseases don't have an effective treatment. Progress is being made, but there is just so much ground to cover in rare disease research. Studying rare diseases…
In early December 2020, mRNA medicines company Arcturus Therapeutics Holdings Inc. ("Arcturus") announced that the company begun dosing participants within its Phase 1b clinical trial. Within the trial, researchers will…
In an article in Medical Xpress, Japanese doctors have performed a “first” by transplanting liver cells (made with embryonic stem cells) into a newborn. The six-day-old infant was diagnosed with…
Ornithine transcarbamylase (OTC) deficiency is a disorder that can cause comas, seizures, and death, making it necessary for those affected to seek treatment. Arcturus Therapeutics aims to give those with…
Three non-investigational New Drug clinical studies are underway for Urea Cycle Disorders (UCD) and various forms of cirrhosis, initiated by Kaleido Biosciences. Dosing has been initiated in each of these…
According to a story from ctvnews.ca, a recent treatment with gene therapy appears to have changed the life of thirty year old Josh McQuillin, who has a urea cycle disorder,…
Urea Cycle Disorders (UCD) are rare genetic conditions caused by an enzyme deficiency. The urea cycle is responsible for removing excess levels of ammonia from the body. Individuals with UCD…
According to a story from Pharma Times, four new medications were recently approved for coverage on the Scottish NHS by the Scottish Medicines Consortium (SMC). Some of these drugs are…
This year has brought an especially nasty and dangerous strain of the flu. Nobody wants the flu, but contracting the virus while suffering from an underlying condition can be more…
Another successful partnership in the rare disease community has come to fruition, reports The Pharma Letter. Just this past week, CureVac and Arcturus Therapeutics will become a powerhouse together with…
G-M-O. Three letters guaranteed to spark lively, if not heated, debate amongst lifelong friends. These three letters, of course, stand for "genetically modified organisms." Why are they so controversial? Why…
Here’s a story that serves as a sobering reminder of how genetic diseases can lay dormant and undetected for years, and how quickly they can come roaring to life… with…
A urea cycle disorder occurs when the body can't break down the ammonia that amino acids produce. Unchecked, ammonia is toxic. According to the National Urea Cycle Disorders Foundation, there…
Ammonia. Even if you don’t know much about it, you know it’s great for cleaning but not so great for ingesting. You… you do know not to ingest it, right?…
Loss is a terrible thing. It’s heavy and overwhelming, and for me, it’s sometimes such a weight that it can be hard to lift yourself up in the morning. Yet,…
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