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gene therapy

News

Breakthrough status for BioMarin ratchets up haemophilia A competition

Breakthrough status for BioMarin ratchets up haemophilia A c...

The FDA has granted Breakthrough Therapy Designation to BioMarin’s gene therapy for haemophilia A, helping to intensify competition in the field.

Views & Analysis

eye

Out of the darkness: new gene therapies for eye diseases

US FDA expert committee’s ‘yes’ could pave the way for a new generation of gene therapies for rare inherited eye disorders.

Views & Analysis

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Addressing market access obstacles for rare bleeding disorde...

Scrutiniseing the market access situation for people with rare bleeding disorders.

News

DNA sequence

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

Biotech has been in talks with US and EU regulators

News

Health wearables

Spark's groundbreaking gene therapy on cusp of US approval

Luxturna wins unanimous backing from FDA advisers

News

eye

FDA experts to vote on Spark's groundbreaking gene therapy

Luxturna could be single life-changing injection

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Editor's Picks

UK and US agree zero tariffs on pharma – with price rises

FDA clears trial of 'vectorised' ALS antibody from VectorY

Tracy Beth Hoeg will be fifth CDER head this year

FDA takes steps to limit non-human primate testing

FDA could hike user fees for ex-US drug programmes

News

CDC panel votes to scrap hepatitis B birth dose advice

CDC panel votes to scrap hepatitis B birth dose advice

CDC advisers vote to overturn decades of US policy and abandon endorsement of universal hepatitis B vaccination for newborns in the US.

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Sales & Marketing

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A pharma company’s core mission is to improve patient outcomes. This hinges on effectively influencing HCP clinical behaviour and driving disease education.

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The rights of trademark owners are acknowledged. Website and content copyright © 2009-2025, pharmaphorum media limited or its licensors; all rights reserved. pharmaphorum – an EVERSANA company