STAT

Gene therapy spurs improvement in rare neuromuscular disease

Kids and teenagers with spinal muscular atrophy had more mobility after receiving a single-dose gene replacement therapy, ...
NJBIZ

FDA approves Novartis’ Itvisma gene therapy for SMA

Novartis’ Itvisma wins FDA approval as the first gene replacement therapy for spinal muscular atrophy (SMA) patients age 2 ...

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. Three-year-old Oliver Chu was born with a rare, genetic condition called Hunter syndrome ...
BioSpace

Novartis Expands Spinal Muscular Atrophy Gene Therapy Market With FDA’s Itvisma Greenlight

The FDA approved an intrathecal form of Novartis’ spinal muscular atrophy gene therapy Zolgensma on Monday, broadening access ...
Fierce Pharma

ASH: CSL proves efficacy of hemophilia B gene therapy Hemgenix holds up after 5 years

Three years after introducing Hemgenix, the first gene therapy for hemophilia B, CSL Behring has released long-term data ...

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 ...
Fierce Biotech

Regeneron makes $150M bet on Tessera's rare disease gene writing prospect

Regeneron is adding its name to Tessera Therapeutics’ unique in vivo gene writing program, inking a deal that lets it in on a ...
Medscape

FDA OKs New Formulation of SMA Gene Therapy Itvisma

Itvisma contains the active ingredient in Zolgensma approved in 2019 for children younger than 2 years but is reformulated ...
PharmaTimes

Five-year study confirms durable efficacy of etranacogene dezaparvovec

Steven Pipe, Professor of Pediatrics and Pathology at the University of Michigan, explained: “The five-year HOPE-B results ...
Samaa TV

World-first gene therapy transforms life of 3-year-old boy

Three-year-old Oliver becomes first Hunter syndrome patient to receive gene therapy, showing remarkable progress a year after treatment.
Hosted on MSN

Columbus doctors turn to gene therapy for patients in continued fight against sickle cell disease

COLUMBUS, Ga. (WRBL) — September is Sickle Cell Awareness Month, and millions of people worldwide are living with the disease. But advances in treatment are being made. WRBL News 3 anchor Cameron ...
EurekAlert!

Pharmacy professor receives $1.9 million award to tackle gene therapy challenges

Assistant Professor of Pharmaceutical Sciences KC Mei standing in his lab at the School of Pharmacy at Binghamton University, State University of New York. BINGHAMTON, N.Y. -- A pharmacy professor at ...