genetic diseases

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Young lonely man in a wheelchair on a balcony looking at the nature in the spring

Catabasis craters after giving up on muscular dystrophy lead

Catabasis has conceded defeat with its Duchenne muscular dystrophy drug edasalonexent, pulling the plug on the drug after a phase 3 trial echoed the results of a failed mid-stage study.

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EMA starts rapid review of Bluebird’s gene therapy for rare ...

Bluebird bio could be just a few months away from approval of its gene therapy for rare disease cerebral adrenoleukodystrophy (CALD) in the EU, after the EMA started an accelerated review.<

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Life with multiple myeloma

Multiple myeloma patient Bob Munro (pictured below) explains what it's like to live with the rare blood cancer and how his diagnosis led him on the journey of a lifetime – cycling from

Catabasis craters after giving up on muscular dystrophy lead

EMA starts rapid review of Bluebird’s gene therapy for rare ...

Life with multiple myeloma

ASCO 2026: Chinese firms take a front seat in oncology, with...

Discover a new era of healthcare panel engagement: Grounded ...

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