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The Latest Breaking News from Gene Therapy

Gene therapy helps combat some forms of blindness – and ongoing clinical trials are looking to extend these treatments to other diseases

An estimated 295 million people suffer from visual impairment globally. Around 43 million of those people are living with blindness. While not every form of blindness can be cured, recent scientific breakthroughs have uncovered new ways to treat some forms of inherited blindness through gene therapy.

The Conversation

'People say we are playing God changing our daughter's genetics - but it saved her'

Nicola Kelleher's partner Bradley Benjamin has a hereditary kidney disease which has seen him suffer on dialysis for years - and they didn't want the same outcome for their child

Daily Mirror

Gene therapy for muscular dystrophy stirs hopes and controversy

The FDA is considering greenlighting the experimental treatment under its accelerated approvals program. Some critics point out the therapy isn't yet proven to work and may be costly.

WEKU

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read the most diverse news in one place.

Genetic therapy can lower tau protein which causes Alzheimer's disease: Study

A world-first trial at UCL and UCLH has found a new gene therapy for Alzheimer's disease that is able to safely lower levels of the harmful tau protein known to cause the disease. Read further on Dynamite News:

Dynamite News

Sarepta Tumbles As Report Of Internal FDA Strife Scorches Its Gene Therapy

Several FDA staffers were leaning toward rejecting a highly watched gene therapy from Sareptas, according to a report Thursday.

Investors Business Daily

Gene therapy: Everything you need to know about the DNA-tweaking treatments

Gene therapies treat or prevent disease by tweaking the content or expression of cells' DNA.

LiveScience

Sarepta Crashes On An Unexpected Roadblock For Its Gene Therapy

In a surprise decision, the FDA will hold an advisory committee to discuss Sarepta's gene therapy, and SRPT stock tanked Friday.

Investors Business Daily

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'Miracle cure' for spinal disorder rolled out on NHS - but babies still face paralysis

A cure for a deadly spinal disorder will be given out on the NHS after pioneering gene therapy costing £1.8million was approved today for infants before symptoms begin

Daily Mirror

'Gene therapy found to wind hearts BACK 10 years and protect people from ageing'

Scientists in Bristol have experimented with the therapy on mice and found it effectively rewound their hearts by the equivalent of 10 human years - soon they hope to use it on heart failure patients

Daily Mirror

Sarepta Rockets And Breaks Out As FDA Smooths The Path For Its Gene Therapy

Sarepta could snag an accelerated approval for its muscular dystrophy gene therapy, analysts suggested Wednesday as SRPT stock rocketed.

Investors Business Daily

Heartbreak as one sibling saved using miracle drug, but older sister not eligible for treatment

Teddi Shaw suffers from deadly metachromatic leukodystrophy and had her stem cells removed and the faulty genes replaced

The Independent UK

Little girl's life saved in Manchester by new gene therapy treatment in UK first

'Without this treatment, we would be facing both our children being taken away'

Manchester Evening News

Shilbottle toddler becomes first child in UK to receive revolutionary gene therapy for fatal disorder

Teddi Shaw, from Shilbottle, suffers from metachromatic leukodystrophy but has been given the chance of a normal life

Chronicle Live

Toddler Teddi is first child in UK to receive revolutionary gene therapy

Young Teddi Shaw will have a normal life thanks to one of the world’s most expensive drugs, but sadly her older sister was too far advanced in her illness to benefit from the treatment

Wales Online

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The Guardian - UK

Girl with deadly inherited condition is cured with gene therapy on NHS

Teddi Shaw, from Northumberland, first recipient on health service of Libmeldy, world’s most expensive drug

The Independent UK

Toddler becomes first child in UK to receive revolutionary gene therapy

Teddi Shaw suffers from deadly metachromatic leukodystrophy (MLD) but has been given the chance of a normal life.

Daily Mirror

'Miracle' gene therapy treatment cures baby facing death - but comes too late for sister

Little Teddi Shaw has become the first NHS patient to receive the treatment for metachromatic leukodystrophy. The 19-month-old was diagnosed with MLD last April

The Independent UK

Promising gene therapy delivers treatment directly to brain

Scientists say delivering gene therapy directly to the brain holds great promise

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The Independent UK

NOT REAL NEWS: A look at what didn't happen this week

Social media users shared a range of false claims this week

Dynamite News

Health: Finds New Infant Gene Therapy To Treat Artemis-SCID Patients

A novel gene therapy developed at UC San Francisco is on track to improve the health of ten young children who were born without functioning immune systems and without the capacity to fight infections. Read on for details:

Reuters

Sickle cell cure will be cost-effective if health disparities considered -study finds

(This Dec.13 story has been corrected to remove reference to Sanofi in the fifth paragraph)

The Guardian - UK

Revolutionary gene therapy offers hope for untreatable cancers

UK teenager is first person in the world to receive ‘base-edited’ cells that could transform cancer medicine

Tribune News Service

World’s most expensive drug approved to treat hemophilia at $3.5 million a dose

U.S. regulators approved CSL Behring’s hemophilia B gene therapy, a one-off infusion that frees patients from regular treatments but costs $3.5 million a dose, making it the most expensive medicine in the world.

The Guardian - US

Gene therapy at $3.5m a dose approved for US adults with hemophilia B

Hemgenix frees patients from repeated treatments but initial cost makes it most expensive medicine in the world

Investors Business Daily

The FDA Just Approved Its Most Expensive Drug Ever — Sending UniQure Stock On A Warpath

The FDA just approved its most expensive drug ever — a hemophilia gene therapy from CSL and UniQure — and QURE stock popped Wednesday.

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