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RNA Interference Therapy "Givosiran" Shows Promise for Treatment of Porphyria

posted by martyb on Monday April 15 2019, @10:22PM   Printer-friendly
from the YADIDNKE-Yet-Another-Disease-I-Did-Not-Know-Existed dept.

takyon writes:

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

Original Submission

 
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  • (Score: 2) by Freeman on Tuesday April 16 2019, @03:23PM

    by Freeman (732) on Tuesday April 16 2019, @03:23PM (#830434) Journal

    Porphyria is a group of diseases in which substances called porphyrins build up, negatively affecting the skin or nervous system.[1] The types that affect the nervous system are also known as acute porphyria, as symptoms are rapid in onset and last a short time.[1] Symptoms of an attack include abdominal pain, chest pain, vomiting, confusion, constipation, fever, high blood pressure, and high heart rate.[1][2][4] The attacks usually last for days to weeks.[2] Complications may include paralysis, low blood sodium levels, and seizures.[4] Attacks may be triggered by alcohol, smoking, hormonal changes, fasting, stress, or certain medications.[2][4] If the skin is affected, blisters or itching may occur with sunlight exposure.[2]

    https://en.wikipedia.org/wiki/Porphyria [wikipedia.org]

    So, some sort of skin disease that can lead to death, but not contagious. That sounds like a whole lot of not fun.

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