Reuters4d
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 ...
Medpage Today on MSN4d
First Brain-Delivered Gene Therapy Approved for AADC Deficiency
The treatment is the first approved gene therapy for AADC deficiency, the FDA said. People with this rare disorder have ...
Alternative experimental gene therapy restores hearing, boosts vision in tests
Harvard Medical School researchers have taken another decisive step in their efforts to develop a gene therapy for people ...
Harvard Medical School2d
Second Gene Therapy Shows Promise for Syndrome Involving Blindness, Deafness
Harvard Medical School researchers have taken another decisive step in their efforts to develop a gene therapy for people ...
Science Daily3d
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.
FiercePharma3d
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 ...
JP Morgan Downgrades Gene Therapy Focused Bluebird Bio, Cites Limited Funding Options
On Thursday, Bluebird Bio Inc. (NASDAQ:BLUE) reported a third-quarter EPS loss of 31 cents, compared to a loss of 80 cents a ...
The American Journal of Managed Care3d
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 ...
Discovery enables effective use of gene therapy for muscular dystrophies and other large-gene diseases
Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is ...
EurekAlert!5d
Mass General Brigham Gene and Cell Therapy Institute names Spark Grant recipients
The Mass General Brigham Gene and Cell Therapy Institute, a hub of innovation dedicated to accelerating groundbreaking research, conducting clinical trials and developing FDA-approved treatments, ...
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 ...
BioPharma Dive3d
PTC wins US approval of gene therapy for fatal enzyme disorder
Kebilidi is the first approved gene therapy that can be directly administered to the brain. Its OK secures a priority review ...