Press release

Screening saves lives: success of screening babies in the UK for rare conditions highlighted on World Rare Disease Day

Public Health England highlights the success of the NHS Newborn Blood Spot Screening Programme in diagnosing babies with rare diseases.

Sasha and Tara Hing

Several babies’ lives are saved every year by screening for the rare, but potentially fatal condition, medium-chain acyl-CoA dehydrogenase deficiency (MCADD).

MCADD makes it hard for the body to break down fats and produce energy and affects 1 in 10,000 babies born in the UK. If left untreated the condition almost always causes severe developmental problems, including serious mental disability or even death.

28 February 2014 marks the 7th annual World Rare Disease Day which aims to raise awareness of rare conditions that can have a serious impact on patients’ lives, such as MCADD.

Sasha Hing was diagnosed with MCADD after she was tested through the NHS Newborn Blood Spot Screening Programme.

Sasha’s mother, Karyn Hing, said:

Sasha was diagnosed following the newborn test. I can recall the phone call from our GP to say that Sasha had something called MCADD. I phoned my husband and was, as you can imagine, quite distressed trying to explain what it was.

Once we had our consultant appointment the next day we had a clearer explanation and felt more in control. We were given a treatment plan and were reassured that our local hospital would be ready in case of any urgent concerns. Our consultant is great and the support offered by The National Information Centre for Metabolic Diseases has been a huge help.

Sasha is a very warm hearted, happy girl. She attends nursery four days a week and as a result of the treatment plan, is able to enjoy a ‘normal’ life.

The newborn screening programme offers screening to all babies in the UK to identify those who are at high risk of having certain serious but rare conditions.

Dr Anne Mackie, Director of Programmes for the UK National Screening Committee (UKNSC), part of Public Health England said;

Last year over 650,000 babies were screened through the programme and 1,287 were identified as high risk for conditions that include sickle cell disease, cystic fibrosis and MCADD.

Screening for MCADD saves approximately 12 children’s lives a year. Early identification, leading to treatment helps ensure these babies can go on to live full and happy lives.

Ends

Notes to editors:

  1. World Rare Disease Day aims to raise awareness amongst the general public and decision-makers about rare diseases and their impact on patients’ lives.
  2. The National Information Centre for Metabolic Diseases is part of the Climb Group. Climb (Children Living with Inherited Metabolic Diseases) is the United Kingdom’s foremost provider of free metabolic disease information to young people, adults, families, professionals and other interested groups.
  3. UKNSC is part of Public Health England.
  4. Public Health England’s mission is to protect and improve the nation’s health and to address inequalities through working with national and local government, the NHS, industry and the voluntary and community sector. PHE is an operationally autonomous executive agency of the Department of Health. www.gov.uk/phe Follow us on Twitter @PHE_uk

Photo courtesy Karyn Hing.

UKHSA press office: screening

Email screeningpressoffice@phe.gov.uk

Phone 020 7654 8120

Out of hours 020 8200 4400

Published 28 February 2014