Discovery enables effective use of gene therapy for muscular dystrophies and other large ...
Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is ...
emjreviews.com17 小时
FDA approves first gene therapy delivered directly to the brain
The therapy, developed by PTC Therapeutics, is designed for patients across the severity of spectrum of AADC deficiency. It ...
Science Daily6 小时
New discovery enables gene therapy for muscular dystrophies, other disorders
StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models.
Pharmabiz5 小时
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., ...
The American Journal of Managed Care18 小时
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 ...
Reuters1 天
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 ...
Business Insider17 小时
PTC Therapeutics Gains Buy Rating Following FDA Approval of Groundbreaking Gene Therapy ...
It is the first gene therapy directly administered to the brain and has received a broad label ... Moreover, the company has been proactive in its commercial launch preparations, identifying centers ...
Monthly Prescribing Reference13 小时
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 ...
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 ...
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 ...
Finanznachrichten1 天
PTC Therapeutics, Inc.: PTC Therapeutics Announces FDA Approval of AADC Deficiency Gene Therapy
Launch preparations are well underway, with centers of excellence already identified and surgeons trained in the procedure to deliver the gene therapy. AADC deficiency is a highly morbid and ...