According to a press release from CTD Holdings, a biotechnology company based in Gainesville, Florida, the Company recently presented data collected from their investigatory Niemann-Pick Disease treatment, Trappsol Cyclo, to families, patients, and anyone else present at the National Niemann-Pick Disease Foundation‘s annual Family Support and Medical Conference. The data comes from two clinical trials of the drug that are still underway.
About Niemann-Pick Type C
Niemann-Pick disease is a condition characterized by abnormalities in a patient’s ability to digest certain fats (like cholesterol) inside the cells. This is due to the absence of the necessary functional enzymes in almost all of the cells in the body.
Niemann-Pick Type C is one of three subtypes of the condition (the others being A and B). The subtypes are similar in some ways, but have significant differences between them. Type C is a genetically-inherited condition, with most cases originating in individuals with family history. Individuals with type B (an often mild form of the condition) may live into adulthood, while Niemann-Pick type A and C patients often experience early mortality.
When cholesterol enters a healthy cell, it is normally metabolized by digestive enzymes that break the fat down and facilitate its exit back through the cell membrane. However, in Niemann-Pick Type C patients, these fats continue to enter and accumulate in cells without ever leaving. The result is fatty deposits in major tissues and organs, including the lungs, liver, or spleen. Eventually, the brain will be affected as well.
As these fats build up, patients may experience physical and mental abnormalities developing. Patients may have trouble eating, swallowing, or walking. They may develop uncontrolled muscle tics and eye movements, and they may be more likely to contract pneumonia.
Currently, no cure for any type of Niemann-Pick exists. Treatments exist for Type C patients, but they are far from a permanent solution — additionally, the treatments are only approved for patients with mild to moderate forms of the disease.
Trappsol Cyclo and the Family Support and Medical Conference
The Family Support and Medical Conference is annually held by the National Niemann-Pick Disease Foundation. In the middle of this month, CTD Holdings gave a presentation on their studies to families and patients. A question and answer segment occurred as well. To ensure that the meeting isn’t just a glorified advertisement, a non-CTD employee hosted the presentation.
Dr. Hrynkow and Dr. Hastings, peers on the research, later joined a panel of other industry and academic representatives to answer audience questions about other type C treatments currently undergoing trial in the United States.
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