Search Results

Treatment of the central nervous system as well as the peripheral organs proves beneficial in severe cases of Spinal Muscular Atrophy (SMA)

A pre-clinical study investigating treatment options for a severe form of Spinal Muscular Atrophy (SMA) has demonstrated that optimal treatment of a morpholino antisense oligonucleotide drug is achieved when the drug reaches the central nervous system as well as the peripheral organs.

Accelerated approval request for a new drug for a rare childhood muscle disorder

A drug for Duchenne Muscular Dystrophy (DMD), originally developed by BRC Theme Lead Professor Francesco Muntoni’s Consortium in the UK, has been filed by Sarepta Therapeutics for accelerated approval by the United States Food and Drug Adminstration (FDA).

Protein responsible for rare childhood disorder gives clues to common neurodegenerative conditions

A new protein that appears to play a role in mitochondrial disease – a rare condition where a lack of energy in cells means that they can’t function properly – could prove to be important in conditions such as Alzheimer’s, Parkinson’s and Huntington’s diseases.

Research at GOSH

Research can improve children’s lives, offering new ways of treating diseases or developing new medicines. This information sheet from Great Ormond Street Hospital (GOSH) explains about the research we do and how to find out more.

Research

The clinical team at GOSH works closely with the research department at the Behavioural and Brain Sciences Unit (BBSU) at UCL Great Ormond Street Institute of Child Health (ICH). We collaborate with colleagues around the world to try and understand the genetic, psychological and neurophysiological basis of disorders on the autism spectrum. All information we obtain in terms of interview, observation and other investigations is compiled into anonymised computerised files. From our database of nearly 2000 children, we are able to compare a child’s problems with others seen in our clinic and can look for similarities and differences. For research purposes, we usually ask parents to donate DNA samples too. 

Research (FDT)

Our current research and development projects include the following:

  • A project to identify the main problem areas and issues related to feeding difficulties in children most relevant to include in parent/carer groups
  • Development and evaluation of a tube weaning protocol
  • Collaboration in a study designed to test how good the DAWBA (Development and Well-Being Assessment) is at identifying problems in children in the community and children seen in clinics (with Prof Tamsin Ford and Prof Robert Goodman)
  • Design and development of a clinical assessment measure for Avoidant Restrictive Food Intake Disorder (with Kamryn Eddy and Jennifer Thomas, Boston, USA)

Research

The Dietetics department works closely with many clinical teams in the hospital where nutrition, growth and development may be an issue.

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