technologynetworks2 小时
Gene Therapy Discovery Enables Use for Muscular Dystrophies and Large-Gene Diseases
A new technology that delivers two halves of a gene separately could enable gene therapy treatments of muscular dystrophies.
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.com20 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 ...
Pharmabiz7 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., ...
Science Daily9 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.
The American Journal of Managed Care20 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 ...
FierceBiotech17 小时
Muscular dystrophy gene therapy uses cells to stitch together mRNA strands in mice
Gene therapy for some diseases, including Duchenne muscular dystrophy (DMD), can be tricky because the needed gene is often ...
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 Insider19 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 ...
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 ...
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 ...