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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

UK budget includes £1bn biopharma investment package

HHS doubles down on controversial peptides plan

Lilly's triple G overpowers Ozempic in diabetes trial

UK puts £100m into "world-first" AI biosecurity facility

US gives $99m for Ebola jab project, as cases spread in Africa

Oncology

On the benefits of earlier oncological intervention, with Johnson & Johnson

On the benefits of earlier oncological intervention, with Jo...

As ESMO 2026 came to a close, pharmaphorum web editor Nicole Raleigh spoke with Johnson & Johnson Innovative Medicine’s Carolyn Sousa and Henar Hevia

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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