The TSC2 arm had an 11% ORR. All patients were heavily pre-treated. The company said the study is expected to be completed by ...
Marinus Pharmaceuticals Announces Key Business Updates for Tuberous Sclerosis Complex Program ...
More information: Jiajia Ye et al, Nutrient deprivation induces mouse embryonic diapause mediated by Gator1 and Tsc2, Development (2024). DOI: 10.1242/dev.202091 ...
TSC is caused by genetic mutations on either the TSC1 or TSC2 gene. A mutation of either of these genes leads to abnormal development and exponential growth of cells within the body. The TSC1 gene ...
Epilepsy is very common in tuberous sclerosis complex and occurs in 80 to 90% of affected individuals during their lifetime. Onset usually occurs during childhood, and up to one third of children ...
The opposite effect was observed in cells that were deficient for the tuberous sclerosis complex (TSC; an endogenous negative regulator of mTOR activity), in which mTOR is hyperactive: Tsc2 − ...
It develops precision therapies for genetically-defined cancer patients with mTOR pathway driver alterations such as alterations in TSC1 or TSC2 genes. The company was founded by Neil Desai in ...
TSC2 Tuberous sclerosis Nucleotide substitution Anxiety, autism, behavioral problems, epilepsy, learning difficulties, mood disorder Hamartomata in multiple organ systems NRXN1 Balanced ...
This is an important milestone as we seek to understand the potential of nab-sirolimus for patients with solid tumors harboring either TSC1 or TSC2 inactivating alterations, a sizable market.