With an end-of-week green light from the FDA, Rocket Pharmaceuticals has officially broken into commercial orbit. | Rocket ...
Morning Overview on MSN
Common gene variant may raise Alzheimer’s risk; researchers eye gene therapy
A genetic variant carried by at least one-fifth of the population may do far more than raise the odds of developing Alzheimer ...
Dr. Donald Kohn has been developing gene therapies for rare pediatric immune disorders for over 30 years. This week, his role ...
Ryuji Morizane, MD, PhD, of the Department of Medicine at Massachusetts General Hospital, is the senior/corresponding author of a new paper published in Signal Transduction and Targeted Therapy, "AAV ...
Shares of Sarepta Therapeutics plunged more than 30% on Friday as the future of its approved gene therapy treatment appeared at risk. The Food and Drug Administration will request that the company ...
The year 2023 marked a significant milestone for the treatment of sickle cell disease (SCD), with the approval of two gene therapies shown to help reduce the pain experienced by people with this ...
MedPage Today on MSN
FDA OKs Therapy for Deadly Pediatric Immune Disorder
Kids with severe LAD-I, caused by ITGB2 mutations, are at risk of life-threatenin ...
The last available hemophilia gene therapy is temporarily unavailable, according to CSL Behring. | The company is working ...
Discusses FDA Approval of KRESLADI Gene Therapy for Severe Leukocyte Adhesion Deficiency Type 1 March 27, 2026 8:30 ...
Nadofaragene firadenovec-vncg is the only FDA-approved nonreplicating intravesical gene therapy for high-risk ...
While the accelerated approval unlocks only a small market opportunity for Rocket Pharmaceuticals, it will give the biotech a ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results