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 has shown “dramatic improvements” following the groundbreaking treatment, his father Ricky Chu said ...
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 Biotech

How a 'healthy exchange of ideas' with RFK Jr. kick-started FDA's gene therapy push

When Tim Hunt was invited to a meeting with Robert F. Kennedy Jr. in early March, the Department of Health and Human Services (HHS) secretary had only been sworn in a few weeks earlier. | When Tim ...
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 ...
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.

New gene therapy approach could reverse ALS, say scientists

Scientists discover a new gene therapy technique that may reverse ALS by restoring vital RNA signals, providing hope for ...
BioProcess International

Leading Ohio children’s hospital expands gene therapy center

The Cincinnati Children’s Center has expanded its capacity to manufacture cellular products for rare diseases.
The American Journal of Managed Care

Understanding Coverage, Financing, and Future Trends of Gene Therapy in Employer Health Plans

The Greater Philadelphia Business Coalition on Health webinar series provided an in-depth framework for the advantages of including cell and gene therapy coverage in employer health plans, as well as ...

India Launches 1st Indigenous, Low-Cost Gene Therapy For Sickle Cell Disease

The world-class, low-cost gene editing solution named "BIRSA 101", in honour of Bhagwan Birsa Munda, remembered as a great ...
MedPage Today on MSN

Gene Therapy Shows Promise for Cutting LDL in Half in Tough-to-Treat Dyslipidemia

NEW ORLEANS – A single injection of a gene-edited protein was deemed productive and safe in patients with dyslipidemia that was unresponsive to lipid-lowering therapy, according to a phase I trial.
Fierce Pharma

Sarepta gets FDA's go-ahead to study immunosuppressive Elevidys regimen in bid to reverse label restriction

When the FDA reworked the prescribing information for Sarepta Therapeutics' Duchenne muscular dystrophy (DMD) gene therapy Elevidys earlier this month, the company touted a plan to study a regimen | ...