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Rare diseases

News

GSK’s ‘bubble baby’ gene therapy passes NICE’s new stricter ...

GlaxoSmithKline’s gene therapy Strimvelis for the treatment of the ultra-rare ‘bubble baby syndrome’ has been approved by NICE.

News

DNA sequence

uniQure presses 'refresh' on haemophilia gene therapy, gets ...

Biotech has been in talks with US and EU regulators

Views & Analysis

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Rare disease market research needs a multifaceted approach

Challenges in rare disease market research and the role of online research.

News

eye

FDA experts to vote on Spark's groundbreaking gene therapy

Luxturna could be single life-changing injection

News

caplacizumab

Ablynx soars after blood clot trial success

European regulators are already reviewing caplacizumab

News

FDA-sign

PTC Therapeutics fails to convince FDA panel about DMD drug

FDA advisers have voted against approval for PTC Therapeutics’ Duchenne drug Translarna because of doubts about its efficacy.

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Editor's Picks

Vertex buys Crinetics for $10bn in endocrinology drive

UK first in Europe to clear Boehringer's IPF drug Jascayd

NICE aims to cut funding for Amgen lung cancer drug Lumykras

Island Pharma drug cleared for Bundibugyo Ebola use

NICE backs NHS use of non-invasive endometriosis tests

News

Dr Anker Lundemose

UK's Mission divests kidney drug to Dimerix for $292m

Mission Therapeutics has struck a deal to out-license a drug for acute kidney injury to Australia's Dimerix, sharpening its focus on CNS diseases.

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Digital

Discover a new era of healthcare panel engagement

Sponsored

Discover a new era of healthcare panel engagement: Grounded ...

A new webinar from Konovo will address some of the critical topics impacting today’s healthcare market research industry.

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The rights of trademark owners are acknowledged. Website and content copyright © 2009-2026, pharmaphorum media limited or its licensors; all rights reserved. pharmaphorum – an EVERSANA company