MedPage Today on MSN1d
Gene Therapy Promising for Rare Genetic Cardiomyopathy
CHICAGO -- Gene therapy for Danon disease, a rare inherited cause of hypertrophic cardiomyopathy, appeared to improve or ...
Reuters6d
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 ...
A new strategy to enhance gene therapy for sickle cell disease
Researchers described a promising new approach for using gene therapy to treat sickle cell disease in the journal Human Gene ...
Promising results from phase 1 gene therapy trial for Danon disease
Researchers at Children's Hospital of Philadelphia (CHOP) announced encouraging results from the first ever gene therapy ...
Rocket Pharmaceuticals' Gene Therapy Progress Boosts Confidence in Future Developments: Analyst
On Monday, Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) presented long-term safety and efficacy results from the Phase 1 study ...
Medpage Today on MSN6d
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 ...
FierceBiotech1d
Neurogene stops Batten gene therapy after FDA denies request
Talks with the FDA haven’t gone according to plan. Seeking a streamlined pathway, Neurogene submitted a Regenerative Medicine ...
BioSpace23h
Neurogene Drops Batten Gene Therapy Program
Failing to receive the RMAT designation from the FDA for its early-stage Batten disease gene therapy, Neurogene tells ...
FierceBiotech2d
CSL Behring shuts down California cell and gene therapy R&D site amid group pivot
In another sign of biopharma's waning interest in the traditional ex vivo gene therapy approach, CSL Behring is shutti | CSL ...
BioSpace1d
Regenxbio Gears Up to Challenge Sarepta in DMD as Gene Therapy Advances to Pivotal Studies
Regenxbio is pushing its Duchenne muscular dystrophy gene therapy into pivotal development, with a BLA planned for ...
中国日报网1d
6 foreign-invested biotech companies enter cell and gene therapy sector in Shanghai
In a significant move towards the liberalization of the healthcare sector, Shanghai's Pudong New Area witnessed the first ...
STAT2d
Regenxbio says gene therapy strengthened boys with Duchenne muscular dystrophy
Based on a small trial with positive muscle performance data, Regenxbio is expected to pursue approval of its gene therapy ...