California boy, 3, receives first-ever gene therapy for his rare disease: ‘It’s just so exciting’

A California toddler is the first person in the world to receive gene therapy to treat his devastating disease.

3-Year-Old Boy with Rare Condition Amazes Doctors by Becoming World's First Gene Therapy Patient

Oliver Chu was born with a rare genetic condition called Hunter syndrome that impacts physical and mental development. The ...

FDA approves Novartis' gene therapy for rare muscle disorder

U.S. Food and Drug Administration has approved Novartis' gene therapy for a type of rare muscle disorder, the drugmaker said ...
New Jersey Business Magazine

Novartis Receives FDA Approval for Spinal Muscular Atrophy Gene Replacement Therapy

The FDA has approved Itvisma for the treatment of children two years and older, teens and adults living with spinal muscular ...

Researchers to test gene therapy for rare diseases with a new FDA trial protocol

Earlier this year, researchers at Children's Hospital of Philadelphia and Penn created a first-of-its-kind drug customized to ...

FDA OKs Novartis SMA Treatment As First Gene Therapy Option For Kids, Teens And Adults

Novartis wins FDA approval for Itvisma, a one-time gene therapy offering broader SMA treatment with sustained motor function ...
AlphaGalileo

GENE THERAPY: A NEW FRONTIER AGAINST GENETIC MUTATIONS, DEGENERATIVE DISEASES, AND CANCERS

A recent review published in Genes & Diseases by researchers from the Naval Medical University and the 922nd Hospital of the Joint Service Support Force of the PLA provides a comprehensive overview of ...
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 ...

She Was Born Without an Immune System. Gene Therapy Saved Her Life.

Born in Morristown, N.J., with virtually no immune system, Cora was diagnosed with severe combined immunodeficiency, a rare ...
News Medical on MSN

Muscle-driven gene therapy shows promise for protecting memory in Alzheimer’s

Alzheimer's disease (AD) is a devastating cause of memory loss and cognitive decline, for which no curative treatment is available.

US FDA approves Novartis' gene therapy for rare muscle disorder

The U.S. Food and Drug Administration has approved Novartis' gene therapy for patients with a rare muscle disorder, the drugmaker said on Monday.