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Toddler doing well after receiving newest gene therapy available on NHS

25 Jun 2025, 9:23 a.m.

A toddler with a life-limiting and life-threatening rare disease is the youngest to be treated with the newest gene therapy available on the NHS at GOSH.

Researcher holding a pipette dropping liquid into a test tube

Genes may help to predict which children will respond well to arthritis treatment

16 Jun 2025, 4:37 p.m.

A team of researchers at Great Ormond Street Hospital (GOSH) and University College London (UCL) have identified a set of genes that could be used to help doctors predict which children will respond well to treatment for juvenile idiopathic arthritis.

Making it easier to invest in frontline care

12 Jun 2025, 4:53 p.m.

GOSH and UCL have signed a landmark agreement to simplify the commercialisation of any joint research and innovation and ensure any financial proceeds are shared equally.

The same cartoon image shown six times with different language subtitles underneath.

Patients and public help create multilingual resources for expectant parents

4 Jun 2025, 3:21 p.m.

A group of clinicians and researchers at GOSH have developed information about a test offered in pregnancy called prenatal sequencing in the UK’s 12 most common languages.

GOSH Arts dance with a child and their parent

NIHR GOSH CRF celebrates International Clinical Trials Day

21 May 2025, 6:09 p.m.

On Tuesday 20 May we celebrated International Clinical Trials Day in NIHR GOSH Clinical Research Facility (CRF).

Computer screen showing dashboard of data

New 'Secure Data Environment' joint offering from GOSH and Aridhia to accelerate clinical research and healthcare innovation

21 May 2025, 2:35 p.m.

We are expanding our partnership with Aridhia to include an offer to external partners.

New VR game to help children rehabilitate after brain tumour treatment

15 May 2025, 1:28 p.m.

The National Institute for Health and Care Research Great Ormond Street Hospital Biomedical Research Centre (NIHR GOSH BRC) is working with partners to develop a new virtual reality (VR) game.

Two men stood in a laboratory, one in a lab coat is receiving his certificate

Healthcare Scientist Award Winners 2025

8 May 2025, 3:57 p.m.

As part of Healthcare Science Week, we hosted an awards ceremony to celebrate our scientists. See the winners for each category below.

Gene therapy offers potential to extend lives of children with rare immune disorder

30 Apr 2025, 10 p.m.

Children have had their lives changed by a ground-breaking gene therapy treatment at Great Ormond Street Hospital for a rare immunodeficiency.

A young girl with short brown hair is smiling in front of a blue sky while sitting in black chair

Cutting-edge genomic technology saves girl from rare brain infection

30 Apr 2025, 8:14 a.m.

Great Ormond Street Hospital has launched the UK’s first accredited ‘metagenomics’ testing service, that allows clinicians to identify infections that are otherwise undetectable.

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.

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).

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.

two women in lab coats stood in front of a table and young child pouring bottle

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!

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.

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.

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.

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.

Man stood up pointing at computer screen with data and woman with blonde hair sat at desk looking at screen

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.

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.