gene therapies

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FDA clears first gene therapy for rare genetic disease WAS

Fondazione Telethon's Waskyra has become the first gene therapy in the US for Wiskott-Aldrich syndrome, and the first taken to market by a non-profit.

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Sarepta backs down and pauses Elevidys shipments

The FDA seems to have won its staring contest with Sarepta over shipments of Elevidys, as the company blinks and agrees to a pause from today.

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A woman holds her hands up in a signal of 'No'

Sarepta resists as FDA seeks pause on Elevidys use

Sarepta has defied an FDA request to halt all use of its gene therapy for Duchenne muscular dystrophy, creating an intriguing regulatory impasse.

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Third death reported with a Sarepta gene therapy

A third fatality has been recorded among patients receiving treatment with a Sarepta gene therapy, adding to the troubles being faced by the company.

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FDA fast-tracks Sanofi's gene therapy for eye disease GA

Sanofi has been granted a speedy review from the FDA for a gene therapy for geographic atrophy, a major cause of blindness.

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FDA probing deaths with DMD gene therapy Elevidys

The FDA has launched a formal investigation into two liver failure-related deaths in patients treated with Duchenne gene therapy Elevidys.

FDA clears first gene therapy for rare genetic disease WAS

Sarepta backs down and pauses Elevidys shipments

Sarepta resists as FDA seeks pause on Elevidys use

Third death reported with a Sarepta gene therapy

FDA fast-tracks Sanofi's gene therapy for eye disease GA

FDA probing deaths with DMD gene therapy Elevidys

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