Rare diseases

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Market Access

Orphan medicines launch excellence: sustaining launch succes...

Overview Launching orphan medicines excellently will become even more important over the next five years as more patients with rare diseases have pharmacotherapies available.

News

abstract shapes, bone structure, 3d render, bone

UK biotech Mereo gears up for key brittle bone drug data

UK biotech Mereo BioPharma is hoping the coming months will transform its fortunes as it prepares for potentially pivotal data for a brittle bone disease drug.

News

NICE backs Akcea’s Tegsedi, pipping Alnylam to hATTR okay

NICE has recommended that Akcea’s hereditary transthyretin-related amyloidosis (hATTR) treatment Tegsedi be made available on the NHS in England, ahead of a rival therapy from Alnylam.

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

Partner Content

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

Partner Content

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.

Orphan medicines launch excellence: sustaining launch succes...

UK biotech Mereo gears up for key brittle bone drug data

NICE backs Akcea’s Tegsedi, pipping Alnylam to hATTR okay

Alnylam to file gene-silencing porphyria drug

Harnessing RWE in rare diseases

3rd Annual Aseptic Processing Summit

NICE backs Roche's non-Hodgkin lymphoma drug Columvi

How to meet your physicians where they are online

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