FDA approves Pfizer’s first gene therapy for rare inherited bleeding disorder
by CNBC
CNBC— The agency greenlighted the drug, which will be marketed as Beqvez, for adults with moderate to severe hemophilia B.
STAT—Young boy dies in trial for Pfizer Duchenne gene therapy. A boy with Duchenne muscular dystrophy died after receiving a Pfizer experimental gene therapy. The company is pausing its trial in older boys.
The Guardian—UK toddler has hearing restored in world first gene therapy trial. Opal Sandy can hear almost perfectly after groundbreaking surgery that took just 16 minutesA British toddler has had her hearing restored after becoming the first person in the world to take part in a pioneering gene therapy trial, in a development that doctors say marks a new era in treating deafness.Opal Sandy was born unable to hear anything due to auditory neuropathy, a condition that disrupts nerve impulses travelling from the inner ear to the brain and can be caused by a faulty gene....
The Independent—Deaf girl is cured and can hear again after first ever gene therapy trial. Opal Sandy, aged 18 months, was born completely deaf due to condition auditory neuropathy