If you are a journalist and would like further information about Pitt Hopkins UK or would like to schedule any interviews, please contact:

Sue Routledge
T: +44 2032392123
E: sue@pitthopkins.org.uk
Website: https://pitthopkins.org.uk/
Facebook: https://www.facebook.com/PitthopkinsUK/
Twitter: @PittHopkinsUK
#pitthopkinsuk
Pitt Hopkins UK is a Registered Charity, Number: 1167153

WHAT IS PITT-HOPKINS SYNDROME? 

Pitt-Hopkins syndrome (PTHS) is a rare neurodevelopmental condition which causes delayed motor development and moderate to severe intellectual disability. Physical symptoms may include severe constipation, and episodes of daytime apnoea (when breathing stops) and hyperventilation. There may be epilepsy. As babies, they will be late reaching developmental milestones and may have feeding problems.

PTHS affects all races and both genders equally. Most cases are “de novo” which means they occur at conception and are not passed on from the parents.

Management of PTHS requires a multidisciplinary team specializing in the care of children with cognitive and motor impairment, including physiotherapists, speech therapists, occupational therapists and others.

WHAT ARE CHILDREN WITH PTHS LIKE?

They are usually moving and busy. Many flap their hands when they are excited. They are normally happy and cheerful and can have bouts of laughter with no clear reason. Most children with PTHS will not learn to speak, although some say a few words and a few children learn to speak quite well. With help many learn to communicate with sign language and pictures.

HOW RARE IS PITT-HOPKINS SYNDROME?

It’s thought that the number of babies born with PTHS could be in the region of 1 in 250,000. It has been estimated that there are around 300 diagnosed cases of Pitt-Hopkins Syndrome in the UK, and at least 1,500 diagnosed cases of Pitt-Hopkins throughout Europe. But many more are still undiagnosed.

The first cases of Pitt-Hopkins Syndrome were identified in 1978. By 2007 only 8 people had been diagnosed with the condition. In 2007 when it was known the condition was caused by a mutation of the gene known as TCF4, a blood test was developed to confirm suspected cases. Since then many more have been diagnosed.

PITT HOPKINS UK

Pitt Hopkins UK has been a registered charity since May 2016. We have six trustees all of whom have children with Pitt-Hopkins syndrome.  We first came into contact as part of the PTHS Google international support group, which was started in April 2008 with a few families from the Netherlands, the UK and the US.

Our goals as a charity are

  • to raise awareness among health professionals to reach earlier diagnosis of Pitt-Hopkins syndrome;
  • to reach other parents of children with PTHS via our social media. We so far have contact with over 80 families, so there are many more diagnosed and undiagnosed who may be managing the condition alone;
  • to support and advocate for parents while delivering the latest information to families;
  • to encourage the  much needed research into the breathing anomalies and the dysfunctional autonomic system which interfere with the quality of life of those with PTHS. 

SHARING INFORMATION

Management guidelines for PTHS have been developed under the guidance of Professor Raoul Hennekam, of the Department of Paediatrics, Academic Medical Centre, Amsterdam, The Netherlands. These are available at https://doi.org/10.1111/cge.13506
Parent-friendly guidelines are being prepared at the moment and we will share these with parents as they become available via our website https://pitthopkins.org.uk/, Google Groups, Facebook groups, Facebook pages, and RareConnect (the international online rare diseases community https://www.rareconnect.org/en).

We have developed a free app for smartphone and tablet, with information and social media links, that is useful for parents, carers, teachers, friends and family of people with Pitt-Hopkins syndrome. Download it from your app store.

We also have a video in English kindly shared with us by the Dutch Pitt-Hopkins charity, which can be viewed here.

International Pitt-Hopkins Awareness Day is on 18th September each year – put it in your calendar!