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Monday 6 April 2026

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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

Marketing - vector modern flat design illustration with mobile device, hand with magnifying glass, darts

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

Novartis snaps up PI3Kα breast cancer drug in $3bn deal

US court says pharma giants must face False Claims suit

Meningitis outbreak in UK prompts call to widen vaccination

US judge rules Kennedy's vaccine changes 'unlawful'

Generic semaglutide in India, and other weight-loss news

Sales & Marketing

Interview with Craig Ackerman Reuters at Pharma USA 2026

Biotech is back, with Craig Ackerman

In Philadelphia this month, pharmaphorum spoke with Craig Ackerman, a partner at the Alexander Group, about launches, AI best practices, and more.

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Sales & Marketing

Using AI-driven synthetic personas to take your insights further

Sponsored

Using AI-driven synthetic personas to take your insights fur...

As AI continues to reshape the healthcare landscape, pharma teams are beginning to leverage synthetic personas.

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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