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Thursday 25 April 2024

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Pompe disease

News

Nexviazyme_Sanofi

Sanofi extends its Pompe range with first Nexviazyme OK

Sanofi has added another string to its Pompe disease therapy bow, after getting FDA approval for Nexviazyme, its latest therapy for the rare, inherited disorder.

News

AstraZeneca (AZ)

Boost for AZ as Ultomiris gets CHMP nod for expanded label

Fresh from its takeover of Alexion, AstraZeneca has picked up a recommendation in the EU for an expansion of the label of Ultomiris, one of the main assets behind the

News

Sanofi Headquarters in Paris

FDA to expedite development of two Sanofi rare disease drugs

The FDA has agreed to expedite development of two rare disease drugs from Sanofi.

News

trumpspeech

Thanks Mr President, but you’re wrong about FDA, says rare d...

NORD patient advocacy group responds to Congress speech

Editor's Picks

Ipsen signs $1.8bn RNA drug alliance with Skyhawk

Metsera lands with $290m to take on obesity giants

Regeneron launches $500m venture capital arm

Telehealth firm Cerebral faces $7m FTC fine over privacy

GSK trails after Pfizer in 5-in-1 meningococcal vaccine race

R&D

pharmaphorum podcast episode 122

Is precision psychiatry possible?

Anyone who has taken a psychiatric medication knows that the status quo in prescribing is a trial and error approach, with patients often cycling through an array of drugs to find the one t

Digital

pharmaphorum podcast episode 121

Fixing clinical trial data management

It’s 2024, and in most clinical trials, data is still being moved from the EHR to the EDC manually – with employees reading data from one screen and typing it onto another.

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The rights of trademark owners are acknowledged. Website and content copyright © 2009-2024, pharmaphorum media limited or its licensors; all rights reserved. pharmaphorum – a Healthware Group company