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Patisiran ph 2 open-label extension study in familial amyloidotic polyneuropathy
Conclusion Data demonstrates that 18-months of patisiran administration was generally well tolerated, resulted in sustained mean serum TTR lowering, supporting the hypothesis that TTR knockdown potentially halts neuropathy progression.
Source: Journal of Neurology, Neurosurgery and Psychiatry - November 14, 2016 Category: Neurosurgery Authors: Schmidt, H., Adams, D., Coelho, T., Conceicao, I., Waddington-Cruz, M., Buades, J., Campistol, J., Pouget, J., Gollob, J., Suhr, O. Tags: Genetics, Neuromuscular disease, Peripheral nerve disease ABN Annual Meeting, 17-19 May 2016, The Brighton Centre, Brighton Source Type: research

Phase 2 open-label extension study of patisiran, an investigational rnai therapeutic for the treatment of familial amyloid polyneuropathy
Familial amyloid polyneuropathy (FAP) is a progressive disease. Patisiran is an investigational small interfering RNA (siRNA) targeting TTR. The primary objective of the Phase 2 study is to evaluate the safety of 0.3 mg/kg patisiran administered intravenously once every 3 weeks. Twenty-seven patients were enrolled; the mean duration of treatment was 7 months (range 3–12), with 282 doses administered (median of 11 doses/patient). Chronic dosing with patisiran has been generally well tolerated. Two patients experienced serious adverse events regarded as being unrelated to study drug. Infusion-related reactions wer...
Source: Journal of Neurology, Neurosurgery and Psychiatry - October 14, 2015 Category: Neurosurgery Authors: Adams, D., Suhr, O., Conceicao, I., Waddington-Cruz, M., Schmidt, H., Buades, J., Campistol, J., Pouget, J., Berk, J., Coelho, T. Tags: Genetics, Neuromuscular disease, Peripheral nerve disease Wed 20, Parallel session 4: Disease pathophysiology Source Type: research