Pre-screening for Spinal Muscular Atrophy could change lives
7 Jan 2022, 6 a.m.
A team from Great Ormond Street Hospital (GOSH) and the UCL GOS Institute of Child Health (ICH) have shown how ‘blood spot’ testing new-borns could identify children who will go on to develop Spinal Muscular Atrophy, (SMA).
Neuromyelitis optica spectrum disorder (NMOSD) is a rare neurological (brain) condition characterised by episodes of optic neuritis (inflammation or swelling of the optic nerve), transverse myelitis (inflammation or swelling of the spinal cord), together
A lumbar puncture is a medical procedure carried out to access the cerebrospinal fluid (CSF). CSF is a watery liquid that surrounds the brain and spinal cord, acting as a ‘cushion’. It also supplies nutrients to the brain. This page from Great Ormond Stre
Multiple sclerosis (MS) is a common neurological condition affecting around 100,000 adults in the UK, but it is rare in childhood – and particularly rare in children under 12. This page from Great Ormond Street Hospital (GOSH) describes the symptoms, caus
ICH researchers develop a technique to 'print' healthy new tissue
24 Jun 2020, 12:51 p.m.
A pioneering international study led by the UCL Great Ormond Street Institute of Child Health (ICH) has seen researchers develop a photosensitive bio-gel that uses light treatment to ‘print’ healthy new tissue directly into specific tissues and organs, an
To celebrate Rare Disease Day we invited patients and their families to try out 13 different hands-on and interactive fun and educational science and research activities for children and adults.
Secretary of State for Health and Social Care visits GOSH
30 Mar 2022, 1:40 p.m.
On Tuesday 29 March, Rt Hon Sajid Javid MP Secretary of State for Health and Social Care, visited our hospital to host a roundtable discussion on rare diseases.
Clinical services at the Dubowitz Neuromuscular Centre
The Dubowitz Neuromuscular Centre provides a comprehensive multidisciplinary assessment, diagnostic and management service for children with neuromuscular disorders.
GOSH celebrates Rare Disease Day 2026 with local community
16 Mar 2026, 11:25 a.m.
GOSH specialises in rare disease research and to celebrate, we recently invited two local North London Year 4 classes to join us for the day to learn about how we care for patients with rare diseases.
Positive interim analysis of Phase 3 studies in spinal muscular atrophy
14 Dec 2016, 10:02 a.m.
Biogen and Ionis Pharmaceuticals announce positive results at interim analysis of two Phase 3 studies both trialling the drug nusinersen in patients with spinal muscular atrophy (SMA).
This information from Great Ormond Street Hospital (GOSH) explains about Myelin Oligodendrocyte Glycoprotein (MOG) antibodies and the symptoms and treatment of MOG antibody associated demyelination.
Methotrexate is a chemotherapy drug that is used to treat certain types of cancer and leukaemia. When given intrathecally, it prevents leukaemia cells entering the cerebrospinal fluid (CSF) around the spine and brain. It is also used to treat leukaemia fo
During Paul O’Grady’s Little Heroes series two, we’re shining a spotlight on the inspiring staff across the hospital who help children get better and fulfil their potential. Consultant spinal surgeon Mark Harris treated patient Reagan, who features on epi
Bowel washouts using an antegrade colonic enema (ACE)
Bowel washouts are a method of dealing with constipation or with soiling, which is the leakage of faeces (poo) other than during a bowel movement. If other methods fail, doctors may recommend bowel washouts using an antegrade colonic enema (ACE). This can
Acute flaccid myelitis (AFM) is a rare condition that tends to occur in ‘outbreaks’ every few years. It affects the spinal cord – particularly the grey matter – and over hours to days muscles weaken and normal limb reflexes disappear. It often follows a m
This page explains about cathererisation using a Mitrofanoff and what to expect when your child comes to Great Ormond Street Hospital (GOSH) to have this procedure.
Spinraza: the first treatment for spinal muscular atrophy to receive FDA approval
30 Mar 2017, 11:28 a.m.
At the end of 2016, the American Food and Drug Administration (FDA) granted approval for the use of Spinraza (nusinsersen) for the treatment of spinal muscular atrophy (SMA) in paediatric and adult patients. This is unprecedented for the SMA community wit
Stunning images showcase life-changing research at GOSH
28 Feb 2023, 9 a.m.
A captivating 3D image that is helping researchers to uncover the secrets of childhood cancers is crowned the winner of our 2022/23 image competition ‘A moment of discovery’.
