— Disease indication – neuroprotection in stroke/ischemia with potential for treating other neurodegenerative diseases
Drug format – neurosteroid
Drug class – drug repurposing (25-hydroxycholesterol)
Research stage and Preliminary data:
In vitro – The inventors demonstrated…
— Background: Studying late-onset neurodegenerative disorders using patient-specific neurons is one of the primary goals of precision medicine. The ability to generate human neurons that mimic the neurons of elderly individuals will provide an instrumental platform to model and study adult-onset neuro…
— Background
Gaucher and Krabbe disease are two types of lipid (fat) storage diseases that can result in progressive damage to the nervous system and other organ systems. Together, these diseases affect about 450,000 people worldwide. In both of these diseases, there is an undesirable accumulation of…
— Background: Neurofibromatosis type 1 (NF1) is a common neurological condition affecting roughly 1 in every 2,500 individuals. NF1 is associated with a host of clinical presentations, including malignant tumors and cognitive defects ranging from attention deficits, mental retardation, and autism. The…
— Background: Brain tumors are the most lethal disease known to mankind with approximately 170,000 annual diagnoses and 13,000 deaths attributed to brain cancer. Glioblastoma, the most common and aggressive, has a median survival of approximately 18 months. Chemotherapeutic agents have been approved b…
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