FDA, gene therapy

US FDA approves PTC Therapeutics' gene therapy for ultra-rare disorder
PTC Therapeutics' gene therapy is already approved in the European Union, Great Britain, and Israel. The drugmaker will also get the FDA's priority review voucher, which allows a drug developer to expedite the review process of its product even if it does ...
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 has already been approved to treat AADC deficiency in Europe and the U.K., among other countries,
FDA Approves Gene Therapy to Treat AADC Deficiency
The approval marks the first time gene therapy will be available to treat patients with aromatic I-amino acid decarboxylase (AADC) deficiency.
FDA Grants Conditional Approval For PTC Therapeutics' Gene Therapy As First Ever With Direct Administration In Brain
PTC Therapeutics' Kebilidi gains FDA accelerated approval as the first brain-administered gene therapy in the U.S. for AADC deficiency treatment.
FDA Approves First Gene Therapy for Treatment of Aromatic L-amino Acid Decarboxylase Deficiency
The U.S. Food and Drug Administration approved Kebilidi (eladocagene exuparvovec-tneq), an adeno-associated virus vector-based gene therapy indicated for the treatment of adult and pediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency.
PTC Therapeutics’ AADC deficiency gene therapy granted FDA approval
PTC Therapeutics (PTCT) announced that the FDA accelerated approval of its gene therapy for the treatment of AADC deficiency, the first-ever
PTC Therapeutics wins FDA nod for gene therapy
PTC Therapeutics (NASDAQ:PTCT) has received FDA approval for its gene replacement therapy Kebilidi for the treatment of children and adults with AADC deficiency, a rare genetic disorder that impairs the ability to generate dopamine,
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Repurposed FDA-approved drug could uncover potential treatment for rare genetic disorders
After years of investigating the diseases' underlying mechanisms, the research team has identified an existing FDA-approved ...
US FDA declines full approval for Intercept's liver disease drug
By Bhanvi Satija and Puyaan Singh (Reuters) - The U.S. Food and Drug Administration on Tuesday declined full approval for ...
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FDA Rejects Full Approval of Liver Disease Drug
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FDA Endorses CaaMTech's Psilocin Drug Development For Depression Treatment
FDA supports CaaMTech's CT-4201 psilocin program for depression, advancing psychedelic therapy and clearing key regulatory ...
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FDA approves first gene therapy delivered directly to the brain
“We look forward to bringing this transformational gene therapy to children and adults with AADC deficiency.” The FDA ...
FDA approves Emrosi for rosacea in adults
The U.S. Food and Drug Administration has approved Journey Medical's Emrosi (minocycline hydrochloride) for the treatment of ...
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FDA approves software to turn Appke Airpods into hearing aids
The Food and Drug Administration (FDA) recently gave Apple the green light to use software that may help million of people ...
Abolish the FDA (opinion)
Many seriously ill people die waiting for the FDA to approve drugs that regulators in other advanced countries have already ...