GM1 GangliosidosisWhat is GM1 gangliosidosis?GM1 gangliosidosis is a rare, progressive lysosomal storage disorder that is characterized by the death of neurons in the brain and spinal cord. Some researchers divide…
According to a story from MarketWatch, the biotechnology company Cyclo Therapeutics announced that is has proceeded with the enrollment for its phase 3 clinical trial. This trial will evaluate an…
On July 8, 2021, gene therapy company Lysogene shared that its gene therapy candidate LYS-GM101, for patients with GM1 gangliosidosis, received Fast Track designation from the FDA. Currently, no treatments…
When I think of anxiety, I think of the movie, Gremlins (1984). For those of you who have seen this movie, you know that feeding a gremlin after midnight turns…
As reported in the Chicago Tribune; Jason Matthews spent his 69 years immersed in only the most thrilling of adventures. After three decades in the CIA, Matthews used eloquence and…
Aromatic L-Amino Acid Decarboxylase (AADC) DeficiencyWhat is aromatic l-amino acid decarboxylase deficiency?Aromatic l-amino acid decarboxylase (AADC) deficiency is a rare genetic disorder that occurs when the enzyme l-amino acid decarboxylase…
CureGRIN is investigating a new symptom of GRIN disorders, a group of rare genetic conditions that cause a wide variety of physical and intellectual symptoms. Referred to as neurostorms, this…
Jason Matthews, the author of the award-winning series Red Sparrow and former CIA officer, passed away on April 28th due to corticobasal degeneration. This rare, neurological condition causes the degeneration and…
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