Gene therapy, Born Blind

Gene therapy experiment gives children 'life-changing' sight boost
An experimental trial of gene therapy has helped four toddlers - born with one of the most severe forms of childhood blindness - gain "life-changing improvements" to their sight, according to doctors at Moorfields Eye Hospital in London.
6-Year-Old Boy Who Was Born Blind Gets Vision Partially Restored After ‘Life-Changing’ Gene Therapy
A Connecticut boy is among four children all born with severe childhood blindness who gained “life-changing improvements” to their vision after an experimental trial of gene therapy. Jace was just 2-months-old when his mother DJ and father Brendan noticed problems with his eyesight.
Blind Children See for the First Time Thanks to a Revolutionary Gene Therapy
A groundbreaking gene therapy has restored sight in four young children born with severe blindness due to a rare genetic deficiency. Scientists at UCL and Moorfields Eye Hospital successfully injected healthy copies of the defective gene into the retina,
Blind 6-year-old Connecticut boy sees for the first time after groundbreaking gene therapy
Jace, 4, from Connecticut, was registered legally blind and only just able to distinguish between dark and light before undergoing treatment at Moorfields Eye Hospital in London
Blind Boy Can See After Groundbreaking Gene Therapy
A young boy, by the name of Harvey, is seen before and after he received gene supplement therapy for a rare inherited eye disorder that caused him to be blind. The after results show a miraculous development in his eyesight.
Gene therapy maker bluebird to go private in discounted deal amid cash crunch
Gene therapy maker bluebird bio on Friday agreed to be taken private by Carlyle and SK Capital Partners at a discount, ending ...
What a $2 million per dose gene therapy reveals about drug pricing
Vincent Gaynor remembers, almost to the minute, when he realized his part in birthing the breakthrough gene therapy Zolgensma ...
Gene Therapy: Revolutionizing Medicine By Tackling Diseases At Their Genetic Roots
Gene therapy represents a paradigm shift in modern medicine, offering unprecedented opportunities to combat diseases at their ...
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3 Gene Therapy Stocks With Game-Changing Potential
Gene therapy stocks are transforming healthcare with revolutionary treatments. Therefore, as this sector continues to evolve, ...
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Pfizer discontinues hemophilia treatment Beqvez, emptying its gene therapy portfolio
Pfizer is pulling further away from the gene therapy field with its decision to discontinue hemophilia product Beqvez. | ...
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Pfizer to Discontinue Its Hemophilia B Gene Therapy
Pfizer's marstacimab (Hympavzi), an IgG1 monoclonal antibody that helps promote clotting, remains on the market. The drug was ...
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Inhalable Cystic Fibrosis Gene Therapy 24 Years In The Making Enters Clinical Trials
A new inhalable gene therapy for cystic fibrosis (CF) has entered Phase I clinical trials, a major milestone on the journey ...
Title: bluebird bio Announces Positive Phase 1/2 Clinical Trial Data for Gene Therapy
In recent financial reports submitted to the Securities and Exchange Committee (SEC) in an 8-K filing, bluebird bio, Inc. (NASDAQ: BLUE), a leading biotech company, has provided an update on their ...
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Envision Summit 2025: Beyond the status quo in gene therapy
At the Envision Summit 2025 in San Juan, Puerto Rico, Mike Farkas, MD, discussed gene therapy research for retinal ...
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Medical Minute: Breakthrough treatment helps children with genetic disorder regain vision
A revolutionary gene therapy treatment in London has improved the vision of children suffering from a rare genetic disorder, ...