News Medical

Gene-replacement therapy could be potential treatment option for SMARD1

Spinal muscular atrophy (SMA) is a disease that causes progressive degeneration in the nerve cells that control muscles, thereby causing muscle weakness and eventually death. SMA affects approximately ...
journalgazette

Novartis receives FDA approval for Itvisma®, the only gene replacement therapy for children two years and older, teens, and adults with spinal muscular atrophy (SMA) | Press ...

Novartis has an exclusive, worldwide license with Nationwide Children's Hospital to both the intravenous and intrathecal delivery of adeno-associated virus 9 (AAV9) gene replacement therapy for the ...
FierceBiotech

In major step toward the clinic, Ring’s human viral vector delivers gene therapy to eyes of mice

Ring Therapeutics’ hunt for new human viruses that can deliver gene therapies multiple times in a single patient appears to have landed on a viable candidate, new mouse data have suggested. In a ...
EurekAlert!

New study reveals the curative potential of genome editing approach for genetic deafness

Adeno-associated virus vector containing an adenine base editor provides an effective strategy to treat hereditary hearing loss caused by the R75W mutation. Moreover, this gene editing technology can ...
GEN

AAV Gene Therapy for Maple Syrup Urine Disease Shows Promise

Maple syrup urine disease (MSUD) is a rare genetic inborn error of metabolism characterized by recurrent life-threatening neurologic crises and progressive brain ...
Morningstar

Novartis receives FDA approval for Itvisma®, the only gene replacement therapy for children two years and older, teens, and adults with spinal muscular atrophy (SMA)

EAST HANOVER, N.J., Nov. 24, 2025 /PRNewswire/ -- Novartis today announced that the US Food and Drug Administration (FDA) has approved Itvisma® (onasemnogene abeparvovec-brve) for the treatment of ...