Baby cured of rare epilepsy syndrome after doctors inject missing gene directly into his brain

An eight-month-old infant with rare but severe epilepsy was cured after receiving an experimental gene replacement in a ...

Israeli hospital performs world's first experimental gene therapy for rare genetic epilepsy

The treatment, administered at Schneider Children’s Medical Center of Israel in Petah Tikva, is a major milestone in the ...
GEN

Gene Replacement Therapy Halts Fatal Arrhythmogenic Condition in Mice

A collaboration between researchers at New York University’s Grossman School of Medicine and Rocket Pharmaceuticals has produced preclinical evidence that gene replacement therapy successfully halts ...
Medical Xpress

Breakthrough gene therapy alleviates Dravet syndrome symptoms in mice without side effects

In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form of epilepsy, scientists have developed a new gene replacement therapy in ...

First-in-the-world gene therapy delivers missing gene directly to infant's brain

An 8-month-old infant with severe genetic epilepsy has become the first patient in the world to receive an experimental gene ...
The American Journal of Managed Care

FDA Approves Gene Therapy Onasemnogene Abeparvovec for Broader SMA Population

Onasemnogene abeparvovec-brve is the first gene replacement therapy approved for SMA patients aged 2 years and older, expanding access beyond infants. Phase 3 trials demonstrated significant motor ...
Nasdaq

Novartis' Gene Replacement Therapy For SMA Demonstrates Meaningful Efficacy And Safety Results

(RTTNews) - Novartis AG (NVS), Wednesday, announced the positive results from its Phase III clinical program of investigational gene replacement therapy, intrathecal onasemnogene abeparvovec or OAV101 ...
Nature

RNA-rewriting candidate moves into the clinic

A growing list of editing tools can be used to make small changes to the genetic code, tweaking key nucleotides to address the underlying causes of diseases. But bigger edits are often needed, and ...
Science Daily

Promising new gene therapy approach for genetic heart disease: clinical trials imminent

Researchers have laid the foundation for the development of a gene therapy for the genetic heart disease arrhythmogenic cardiomyopathy (ACM). Their approach, based on replacement of the PKP2 gene, led ...
Nasdaq

FDA Greenlights Novartis' Gene Replacement Treatment for SMA

Novartis NVS obtained FDA approval for onasemnogene abeparvovec-brve, a gene replacement therapy, for children two years and older, teens, and adults with spinal muscular atrophy (SMA). The therapy ...