STAT3h
FDA commissioner: We need action and higher-quality research on ultra-processed foods
Researchers, public health and health care groups, advocates, industry, and the public must put tackling diet-related disease ...
Pharmabiz8h
PTC Therapeutics’ gene therapy, Kebilidi to treat AADC deficiency receives US FDA approval
PTC Therapeutics’ gene therapy, Kebilidi to treat AADC deficiency receives US FDA approval: Warren, New Jersey Friday, November 15, 2024, 10:00 Hrs [IST] PTC Therapeutics, Inc., ...
New FDA rules for TV drug ads: Simpler language and no distractions
The new rules, which cover both TV and radio, instruct drugmakers to use simple, consumer-friendly language when describing ...
BioPharma Dive15h
PTC wins US approval of gene therapy for fatal enzyme disorder
Kebilidi is the first approved gene therapy that can be directly administered to the brain. Its OK secures a priority review ...
Monthly Prescribing Reference16h
Kebilidi, a Gene Therapy for AADC Deficiency, Gets Accelerated Approval
The Food and Drug Administration (FDA) has granted accelerated approval to Kebilidi™ (eladocagene exuparvovec-tneq) for the treatment of adult and pediatric patients with aromatic L-amino acid ...
precisionmedicineonline20h
PTC Therapeutics' AADC Deficiency Gene Therapy Kebilidi Gains FDA Accelerated Approval
This is the first gene therapy for AADC deficiency to receive US regulatory approval and the first that can be administered directly to the brain.
FiercePharma21h
PTC wins FDA approval for first brain-delivered gene therapy Kebilidi
The FDA issued its first stamp of approval for a cell or gene therapy back in 2017 to Novartis' Kymriah. | The gene therapy ...
FDA grants accelerated approval for PTC’s AADC deficiency gene therapy
The US Food and Drug Administration (FDA) has granted accelerated approval for PTC Therapeutics’ KEBILIDI, the first gene ...
The Pharma Letter22h
FDA nod for AADC deficiency gene therapy from PTC
The US Food and Drug Administration (FDA) has granted accelerated approval of PTC Therapeutics’ gene therapy for the ...
PTC Therapeutics wins FDA nod for gene therapy
PTC Therapeutics (PTCT) stock on watch as company receives FDA approval for gene therapy Kebilidi for AADC deficiency. Read ...
pharmaphorum1d
PTC gets FDA okay for first brain-delivered gene therapy
Kebilidi is the first gene therapy approved by the FDA for administration directly into the brain, given via a cannula during ...