Reuters21h
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 ...
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 ...
Repurposed FDA-approved drug could uncover potential treatment for rare genetic disorders
In a new study, researchers at McMaster University have identified a potential treatment for Sandhoff and Tay-Sachs ...
The American Journal of Managed Care4h
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 ...
FiercePharma4h
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 ...
Abolish the FDA (opinion)
Many seriously ill people die waiting for the FDA to approve drugs that regulators in other advanced countries have already ...
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 ...
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 ...
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 ...
News Medical22h
The benefits and challenges of FDA Fast-Track and Accelerated Approval in drug development
A primary benefit of the FDA Fast Track designation is how it accelerates the drug approval process. Early and continuous ...
emjreviews.com4h
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 ...
Cure Today5h
A Phase 3 Trial of Amezalpat in Advanced Liver Cancer Receives Study May Proceed Letter
For unresectable or metastatic liver cancer, the TPST-1120-301 trial of amezalpat combination therapy has received a “Study ...