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Strensiq

News

AZ considers filings for long-acting Strensiq follow-up

AZ considers filings for long-acting Strensiq follow-up

AstraZeneca's long-acting Strensiq follow-up efzimfotase alfa has hit the mark in children with rare disease HPP, but the data in adults is unclear.

News

Strensiq

Alexion lambasts NICE over rare disease ruling

Watchdog accused of ignoring stakeholder consensus and call for negotiations

News

NICE1

Rare disease drug Strensiq too expensive, says NICE

Alexion's new rare disease treatment Strensiq has been rejected by England's health watchdog NICE in draft guidance.

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News

Emil Kakkis

Ultragenyx to slash costs after Angelman trial failure

Ultragenyx's therapy for the rare genetic disorder Angelman syndrome, apazunersen, has flunked a late-stage trial, crashing its share price.

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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