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from the YADIDNKE-Yet-Another-Disease-I-Did-Not-Know-Existed dept.
Promising Results with Givosiran in Acute Hepatic Porphyrias[*]
Investigational givosiran met the primary endpoint of reduction in the annualized rate of composite porphyria attacks versus placebo in patients with acute hepatic porphyria (AHP), researchers said here.
In the interim analysis of the phase III ENVISION trial, patients treated with givosiran experienced a mean composite annualized rate of 3.2 attacks versus 12.5 attacks in patients on placebo (P<0.0001), for a mean reduction of 74%, said Manisha Balwani, MD, of the Mount Sinai School of Medicine in New York City, and colleagues.
"We saw a robust treatment effect," said Balwani at a press conference at the European Association for the Study of the Liver (EASL) annual meeting.
"Currently, there are no approved therapies aimed at preventing the painful, often incapacitating attacks, and chronic symptoms associated with acute hepatic porphyria," she added. "The results from ENVISION are promising and demonstrate a strong treatment effect for givosiran, with reduction of attacks and improvement in patient-reported measures of overall health status and quality of life."
[*] Porphyria.
BBC has a dumbed down article: Gene-silencing: 'New class' of medicine reverses disease porphyria
(Score: 0) by Anonymous Coward on Tuesday April 16 2019, @03:55AM
It is just jargon to make you not realize how stupid they are.
"We gave a drug to people/rats/cells and measured a bunch of crap. A random one of the results can be spun to get desperate people to give us shittons of money if you give us shitloads of money to risk get it past the FDA."
The FDA askes for two "statistically significant" trials. Where statistically significant is defined as p-value < 0.05. Once you go look up what a p-value means and the other data, please respond with how many you expect out of drugs that do nothing every year.