genetic diseases Archives -

Sarepta is pressing forward with a bold plan to file with the FDA for accelerated approval of its

Sarepta says early filing for DMD gene therapy is back on

https://pharmaphorum.com/news/sarepta-says-early-filing-for-dmd-gene-therapy-is-back-on/

Acadia Pharma’s trofinetide has hit its efficacy targets in a phase 3 trial involving girls with the devastating

Acadia closer to bringing first Rett syndrome drug to market

https://pharmaphorum.com/news/acadia-closer-to-bringing-first-rett-syndrome-drug-to-market/

Catabasis has conceded defeat with its Duchenne muscular dystrophy drug edasalonexent, pulling the plug on the drug after

Catabasis craters after giving up on muscular dystrophy lead

https://pharmaphorum.com/news/catabasis-craters-after-giving-up-on-muscular-dystrophy-lead/

Bluebird bio could be just a few months away from approval of its gene therapy for rare disease

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

https://pharmaphorum.com/news/ema-starts-rapid-review-of-bluebirds-gene-therapy-for-rare-disease-cald/

Multiple myeloma patient Bob Munro (pictured below) explains what it’s like to live with the rare blood cancer and

Life with multiple myeloma

https://pharmaphorum.com/partner-content/life-with-multiple-myeloma/

genetic diseases