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The Latest Breaking News from Gene Therapy

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

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 Guardian - 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.

The Independent UK

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'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

Daily Mirror

Promising gene therapy delivers treatment directly to brain

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

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

The Independent UK

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:

Dynamite News

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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)

Reuters

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

The Guardian - UK

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.

Tribune News Service

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

The Guardian - US

$3.5M gene therapy for hemophilia gets FDA approval

U.S. health regulators have approved the first gene therapy for hemophilia, a blood-clotting disorder with few treatment options

The Independent UK

Epilepsy: gene therapy technique targeting overactive brain cells shows promise in treating drug-resistant form of the condition

Something like 50 million people worldwide have epilepsy. While the majority of these people are able to use medications to manage and prevent their seizures, around one-third don’t respond well to these treatments. In such cases, the only option available to bring seizures under control is to remove the part…

The Conversation

Death in CRISPR gene therapy study sparks search for answers

The lone volunteer in a unique study involving a gene-editing technique has died, and those behind the trial are now trying to figure out what killed him

The Independent UK

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Wales Online

Four brothers with rare disease who had bone marrow transplants lead call for more research

There is 'promising new gene therapy treatment on the horizon' that could see 'safer and more effective ways to treat patients with XLP'

Tribune News Service

Bluebird’s gene therapy for brain disease receives accelerated approval

Bluebird Bio Inc.’s gene therapy for a brain-wasting disease received accelerated approval from U.S. regulators, making it the first treatment of its kind for children living with cerebral adrenoleukodystrophy.

Benzinga

FDA Okays bluebird bio's $3M Gene Therapy For Rare Neurological Disorder In Boys: What Investors Should Know

Biotechnology company, bluebird bio, Inc. (NASDAQ: BLUE) announced late Friday that the Food and Drug Administration approved its gene therapy for a rare neurodegenerative disease.

Liverpool Echo

Gene therapy firm growing its Liverpool footprint with new build

A three-storey lab site was approved earlier today

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Benzinga

Recap Of Tuesday's Biotech Catalysts - End Of the Day Summary

Amgen (NASDAQ:AMGN) announced top-line results from the global Phase 3 CodeBreaK 200 trial evaluating once daily oral LUMAKRAS (sotorasib) in patients with KRAS G12C-mutated non-small cell lung cancer (NSCLC), the study met primary endpoint. Amgen shares traded in a range of $238.29 to $240.31 on day volume of 2.43 million shares, closed regular…

Benzinga

Recap Of Wednesday's Biotech Catalysts - End Of the Day Summary

The European Commission (EC) has granted conditional marketing authorization (CMA) to BioMarin Pharmaceutical’s (NASDAQ:BMRN) lead asset ROCTAVIAN (valoctocogene roxaparvovec) gene therapy for the treatment of severe hemophilia A (congenital Factor VIII deficiency) in adult patients. BioMarin shares traded as high as 2.28 percent, in a range of $91.39 to $93.68…

Wales Online

Alzheimer's breakthrough: Scientists reverse disease with gene therapy

In the study, stem cell survival was enhanced by deleting a gene called Bax

The Conversation

Helping cells become better protein factories could improve gene therapies and other treatments – a new technique shows how

The cells in your body are not all the same. Each of your organs has cells with very different functions. For example, liver cells are top-notch secretors, as their job requires them to make and export many of the proteins in your blood. By contrast, muscle cells are tasked with…

Benzinga

Recap of Wednesday's Biotech Catalysts - End of The Day Summary

IDEAYA Biosciences (NASDAQ:IDYA) has entered into a clinical trial collaboration and supply agreement with Amgen Inc. to evaluate the efficacy and safety of IDE397 with Amgen's AMG 193 in a Phase 1 clinical trial. IDEAYA shares traded in a range of $14.59 to $15.82 on a day volume of 130.58…

Manchester Evening News

Mum thought itchy hands were caused by household cleaner - she was left with a deadly diagnosis costing £16.5K a month

Without treatment she now has just three to six months to live, doctors say

The Independent UK

Gene therapy dramatically cuts risk of bleeding in haemophilia B patients

Haemophilia is a rare condition that affects the blood’s ability to clot.

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