SparingVision has raised €75 million in second-round funding that will be used to fund clinical trials of gene therapies for ocular diseases retinitis pigmentosa (RP) and dry age-related ma
Sarepta is pressing forward with a bold plan to file with the FDA for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy (DMD) in the next few months, with a
Acadia Pharma's trofinetide has hit its efficacy targets in a phase 3 trial involving girls with the devastating neurological disorder Rett syndrome, although safety has dampened enthusiasm
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.
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.<
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 Lond
After rejecting ImmunityBio’s Anktiva last year, the FDA has cleared the drug as a treatment for bladder cancer, making it the company’s first commercial-stage product.
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.