Reuters2d
US FDA approves PTC Therapeutics' gene therapy for ultra-rare disorder
Nov 13 (Reuters) - The U.S. Food and Drug Administration approved PTC Therapeutics' (PTCT.O), opens new tab gene therapy to ...
PTC Therapeutics Announces FDA Approval of AADC Deficiency Gene Therapy
PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today the U.S. Food and Drug Administration (FDA) accelerated approval of its gene therapy for the treatment of AADC deficiency, the first-ever gene ...
The Pharma Letter1d
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 ...
GlobalData on MSN1d
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 ...
Pharmabiz1d
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., ...
PTC Therapeutics, Inc.: PTC Therapeutics Announces FDA Approval of AADC Deficiency Gene Therapy
First-ever FDA approval for gene therapy directly administered to the brain - - Priority review voucher granted - - Broad label including children and adults - - ...
Daily13h
FDA Approves First Gene Therapy for Treatment of AADC deficiency
The U.S. Food and Drug Administration approved Kebilidi (eladocagene exuparvovec-tneq), an adeno-associated virus ...
ClearPoint Neuro announces SmartFlow Neuro Cannula FDA authorization
ClearPoint Neuro (CLPT) “announced the U.S. Food and Drug Administration has granted marketing authorization for the SmartFlow Neuro ...
Le Lézard2d
NOTICE TO THE MEDIA - Government of Canada to announce funding to support youth mental wellness
The Honourable Ya'ara Saks, Minister of Mental Health and Addictions and Associate Minister of Health, will host a panel discussion to discuss providing mental health, counselling, and crisis support ...
BioSpace4d
After Decades of Failure, First Disease-Modifying Huntington’s Treatment on the Horizon
The past four years have brought disappointment for the Huntington’s community, but optimism is growing as companies ...
precisionmedicineonline1d
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.
pharmaphorum2d
FDA's Duchenne drug approval greeted with joy, but remains controversial
The US Food and Drug Administration approved ... exchange following the announcement. Shares in rival PTC Therapeutics, which has a DMD drug conditionally approved in the EU but not in the ...