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New research analyses key immune cell

28 Mar 2025, 11:16 a.m.

A new study led by researchers at University College London Great Ormond Street Institute of Child Health has found that women have a higher proportion of key immune cells between puberty and menopause.

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First patient treated with groundbreaking gene therapy trial

21 Mar 2025, 9:51 a.m.

A baby boy born with a rare condition has become the first in the world to be treated with a new, potentially lifechanging, investigational gene therapy on a clinical trial at Great Ormond Street Hospital (GOSH).

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Headshot of Professor Darren Hargrave.

Professor Darren Hargrave appointed NIHR Senior Investigator

19 Mar 2025, 2:44 p.m.

We’re delighted to announce that Professor Darren Hargrave has been named as a NIHR Senior Investigator in this year’s prestigious awards list.

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Reflecting on rare disease day 2025

7 Mar 2025, 12:23 p.m.

Rare Disease Day is an international event which takes place annually on the last day of February. The date is chosen because in leap years it is February 29, the rarest date!

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Imogen as a toddler with her dad

First-of-its-kind study will assess psychological impact of sudden cardiac death on children

3 Mar 2025, 9:36 a.m.

Great Ormond Street Hospital (GOSH) and University College London (UCL) will lead a pioneering study with the hope of revealing more about how best to help children who have experienced an unexpected and often traumatic loss in the family.

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A small girl in a blue full body swimsuit and a straw hat plays in the sand with her back to the camera. She has a blue bucket and the sun is streaming across the floor

Genomics will revolutionise treatment for childhood rare diseases

27 Feb 2025, 10 a.m.

A global study has proven the power of a simple genomics blood test to improve treatment and care for childhood epilepsy.

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Jessica's care team at the GOSH Clinical Research Facility

Rare Disease Day: Jessica's story

26 Feb 2025, 5:14 p.m.

As part of this year’s countdown to Rare Disease Day, we’re sharing Jessica’s story.

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Surgical team at GOSH

Revolutionary eye gene therapy gives children life-changing improvements

21 Feb 2025, 10:31 a.m.

Four children have gained life-changing improvements in their sight following pioneering eye gene therapy at Great Ormond Street Hospital for a rare genetic blindness.

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The important role of data science in accelerating advancements in care for rare diseases

20 Feb 2025, 4 p.m.

For many of young patients, especially those with rare or complex conditions, getting the right diagnosis and treatment can be a challenging journey. That’s where data science can make a real difference.

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Predicting how childhood kidney cancers develop

18 Feb 2025, 2:14 p.m.

New research has analysed how the childhood kidney cancer, Wilms tumour, evolves in children who are more at risk from developing the cancer.

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GOSH Professor appointed to co-chair national children and young people cancer task force

14 Feb 2025, 9 a.m.

We are delighted to congratulate Prof. Darren Hargrave and Dame Caroline Dinenage on their appointments as co-chairs of the Children and Young People Cancer Task force.

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GOSH dietitians celebrate research success

13 Feb 2025, 11:39 a.m.

The dietetics team have had a busy year carrying out research alongside their clinical practice.

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Join us in the countdown to Rare Disease Day 2025

1 Feb 2025, 9 a.m.

Rare Disease Day takes place annually on the last day of February and is a global opportunity to increase awareness and advocate for those living with rare conditions.

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Staff member walking down hospital corridor

Significant rise in mental health admissions for young people over last decade

23 Jan 2025, 1:48 p.m.

The first study of its kind has found a 65% increase in the number of children and young people admitted to general acute medical wards in hospitals in England, because of a mental health concern between 2012 and 2022.

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Millions in new funding for revolutionary paediatric intensive care trial

16 Jan 2025, 9:32 a.m.

Researchers at Great Ormond Street Hospital (GOSH) have received more than £6m in funding from the National Institute for Health and Care Research (NIHR).

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Professor Ajay Vora awarded MBE in King’s New Year’s Honours

31 Dec 2024, 11:55 a.m.

Professor Ajay Vora has been awarded an MBE in the New Year’s Honours List

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Twelve days of Research and Innovation at GOSH

23 Dec 2024, 1:51 p.m.

It’s been a busy year for Research and Innovation at GOSH, with a number of ground breaking research trials, advanced data projects and technology pilots. To round off the year, here are just twelve of the stories (and amazing staff and researchers) that

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NIHR GOSH Clinical Research Facility celebrates patients and their families with a festive celebration

20 Dec 2024, 1:21 p.m.

On Wednesday 18 December we celebrated the festive period at the National Institute for Health Research (NIHR) GOSH Clinical Research Facility (CRF) on the 8th floor of the Southwood building.

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Six spherical bundles of cells with complex networks are pictured, in two lines of three. These are (from left to right) gold, purple and green (top left), purple, green and yellow (bottom left), red and gold (top centre), blue and orange (bottom centre), green, purple and blue (top right), and yellow, blue and orange (bottom right).

Duchenne Muscular Dystrophy gene therapy trial highlights complexity of disease

19 Dec 2024, 5:04 p.m.

The first large-scale trial of gene therapy for the debilitating neuromuscular disease, Duchenne Muscular Dystrophy (DMD) has been carried out

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New findings from world’s largest study on children with Long-Covid

17 Dec 2024, 4:36 p.m.

A new study led by clinicians and researchers at Great Ormond Street Hospital and University College London Great Ormond Street Institute of Child Health has found that 70% of young people in England with Long-Covid recover within two years.

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