Porphyria is a group of diseases in which substances called por... - dofaq.com
porphyria

Porphyria is a group of diseases in which substances called por...

wikipedia - 07 Nov 2016
Porphyria is a group of diseases in which substances called porphyrins build up, affecting the skin or nervous system. The types that affect the nervous system are also known as acute porphyria. Symptoms of acute porphyria include abdominal pain, chest pain, vomiting, confusion, constipation, fever, and seizures. These symptoms typically come and go with attacks that last for days to weeks. Attacks may be triggered by alcohol, smoking, stress, or certain medications. If the skin is affected, blisters
porphyria

Alnylam drops early filing plan for porphyria drug givosiran

FierceBiotech - 15 Oct 2018
The gene-silencing specialist revealed last month that it was considering regulatory submissions based on an early readout of biomarker data from the ENVISION trial, but after discussions with the FDA now says it will wait for data on porphyria attack ...
porphyria

Antifungal agent found to be possible treatment for porphyria

Medical Xpress - 24 Sep 2018
A large team of researchers from Spain, France and the U.S. has found that a common antifungal agent might be useful as a treatment for a rare type of porphyria. In their paper published in the journal Science Translational Medicine, the group ...
porphyria

Existing Drug Could Treat Porphyria

Technology Networks - 19 Sep 2018
An existing antifungal drug, ciclopirox (CPX), has been shown to improve symptoms associated with congenital erythropoietic porphyria – the rarest type of porphyria. The study was conducted using a mouse model of the disease, the team's findings were ...
porphyria

Hepatic porphyria treatment achieves topline interim results

Healio - 27 Sep 2018
Alnylam Pharmaceuticals announced positive topline results from an interim analysis of the Envision phase 3 study of givosiran, an RNA interference therapeutic designed to target aminolevulinic acid synthase 1, for the treatment of acute hepatic ...
porphyria

Alnylam to Pursue Full Approval & Rolling NDA for Givosiran

Nasdaq - 16 Oct 2018
The results showed that treatment with givosiran led to a statistically significant reduction in urinary aminolevulinic acid (ALA) levels in acute intermittent porphyria (AIP) patients compared with placebo. ALA is a disease biomarker, which is likely ...

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