Rare diseases

News

Alnylam to file gene-silencing porphyria drug

Alnylam is to file its givosiran with regulators to treat a form of the blood disease porphyria, applying its gene silencing technology to the condition for the first time, following suppor

Views & Analysis

Harnessing RWE in rare diseases

Ahead of eyeforpharma Philadelphia 2019, Sonal Bhatia, Pfizer’s vice president, North America medical lead, rare disease talked to us about the importance of collaboration and RWE to he

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$Banner for Media partners {460x250px}_3rd Aseptic Processing$

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3rd Annual Aseptic Processing Summit

Vonlanthen Group is pleased to invite you to the 3rd Annual Aseptic Processing Summit scheduled on November 20-21, in Vienna, Austria.

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$Banner for Media partners {460x250px}_3rd Genotoxic$

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3rd Annual Impurities: Genotoxic and Beyond Summit

We are pleased to invite you to the 3rd Annual Impurities: Genotoxic and Beyond Summit scheduled for June 12th-13th, 2019.

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$Banner for Media partners {460x250px}_Gene and Cell Therapy$

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Gene and Cell Therapy: Quality Developments to Commercializa...

We are pleased to invite you to the Annual Gene and Cell Therapy: Quality Developments to Commercialization Summit, scheduled for June 5-6, 2019, in Berlin, Germany.

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