BioNews
FDA (Food and Drug Administration)
The Food and Drug administration is a US federal agency responsible for protecting public health. It is the body responsible for regulation and approval of drugs and other pharmaceutical products such as vaccines within the USA.
First single-dose haemophilia B gene therapy approved by FDA
An adeno-associated virus vector-based gene therapy, Hemgenix, has been approved for adults with Haemophilia B by the US Food and Drug Administration...
Concern over court ruling against the FDA regulating cell and gene therapies
The recent court ruling that the US FDA's powers do not extend to regulating some stem cell treatments is a blow for those hoping for better regulation of unproven treatments, argues Dr Patrick Foong...
Drug silences motor neurone disease mutation
Tofersen was effective in slowing the progression of a rare form of motor neurone disease when taken over a period of 12 months...
FDA recommends additional confirmatory testing alongside NIPT
Prenatal genetic test results need to be communicated better, according to the US Food and Drug Administration...
New gene therapy for patients with blistering skin disorder
Patients with a rare genetic skin condition have been successfully treated, for the first time, using a novel gene therapy...