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 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).
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.
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 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.
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.
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.
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.
Young people donate tissue samples to unlock mysteries of arthritis
2 Jul 2025, 9:49 a.m.
A new groundbreaking study led by researchers at the University of Birmingham, Great Ormond Street Hospital, University College London and Birmingham Children’s Hospital has revealed important clues into what is driving arthritis in children.
Stem cell treatment can improve rare skin condition in children
15 Aug 2025, 9:46 a.m.
Children with a rare skin condition have found their symptoms have improved through taking part in the largest clinical trial of its kind, led by clinicians at Great Ormond Street Hospital for Children (GOSH).
Nanodiamonds and hormones used in rare condition to promote lung growth
27 Aug 2025, 9 a.m.
An international research team led by GOSH, UCL and KU Leuven in Belgium, is using 3D-printing and nanodiamonds, to design treatments that could help babies repair their damaged lungs in the womb.
What do bush babies, tamarin monkeys, and mouse lemurs have that humans don’t?
28 Aug 2025, 10:41 a.m.
GOSH imaging researchers have worked with teams in Harvard in the USA to work out how and when our pelvises developed to be different from other animals.
Scientists discover clues to help children with rare muscle disease
8 Sep 2025, 4:20 p.m.
New ground-breaking research by experts at Great Ormond Street Hospital (GOSH) and University College London (UCL) has led to an exciting discovery that could help children with the rare muscle disease, juvenile dermatomyositis (JDM).
New study pinpoints why some transplanted kidneys are rejected
17 Sep 2025, 8:44 p.m.
Researchers at University College London (UCL), the Wellcome Sanger Institute and the University of Cambridge combined technology that determines the genetic or RNA sequence of individual cells with powerful 3D imaging to look at lymphatic vessels
Landmark gene therapy study shows safety for children
16 Oct 2025, 9:54 a.m.
Results from the largest cohort of children who received a gene therapy for a rare immunodeficiency condition have shown the long-term safety and efficacy of the curative treatment.
