Useful links
Below we have listed some of the many organisations and partners which you may find useful. If there are any others you can think of that we may have missed then email us.
Although Amicus Therapeutics are not conducting any research into MLD therapies, they produce an excellent online booklet called ‘What’s missing?’ which gives a generalised description of the processes which are at fault in Lysosomal Storage disorders. www.amicustherapeutics.com
Brains for Brain Foundation are the organising body behind the European Task Force on Brain and Neurodegenerative Lysosomal Storage Diseases. They bring together a consortium of some 80 different clinicians and research scientists who are investigating this group of disorders and seeking therapeutic treatments. www.brains4brain.eu
Cauldwell Children provides family support services, equipment, treatment and therapies for disabled children and their families across the U.K. www.caudwellchildren.com
The Counselling Directory is a confidential service that encourage those in distress to seek help, with links to accredited counsellors throughout Britain. www.counselling-directory.org.uk
Disability Grants helps individuals, families and carers supporting people with a disability, to find grants and funding towards the cost of disability equipment, holidays, housing, days out and many other things above and beyond the normal costs of everyday living. www.disability-grants.org
Harry’s Rainbow supports children and young people in the Milton Keynes and surrounding areas, who have been bereaved of a parent or sibling. Giving information and guidance on their website, they also provide various services which include support groups, activities and trips, and individual tailored support. www.harrysrainbow.co.uk
The Jessie May Trust in Bristol CHSW (children’s hospice), is a charity that provides hospice at home care for terminally ill children in Bristol and the South West. The charity was set up by the parents of Jessica May Purrington, a little girl who died at home aged 4 months, surrounded by her family and her nurses. Jessie May has a team of dedicated nurses who provide care to children and families during a child’s life and after their death. www.jessiemay.org.uk
Thousands of disabled and terminally ill children in the UK rely on Newlife to get the equipment they need that has often been refused by the statutory services or because of delays in provision. Newlife runs the only fast track equipment services in the UK helping those children in urgent need. www.newlifecharity.co.uk
As the East Midlands only hospice for children and young people, Rainbows is a place where life limited children and their families can find care and support. An incredible team of people help relieve symptoms, improve quality of life, care for children at the end of life and support parent and sibling through their bereavement. www.rainbows.co.uk
The Rainbow Trust Children’s Charity provides emotional and practical support to families who have a child with a life threatening or terminal illness. www.rainbowtrust.org.uk
Roald Dahl’s Marvellous Children’s Charity exist to make seriously ill children’s lives better, by providing nursing care, equipment, carers or toys. www.roalddahlcharity.org
ROMPA was created to improve the life quality of people of all ages and all abilities through unique sensory products and meticulously installed environments. They pride themselves on excellent customer service in order to meet the needs of individuals, institutions and businesses globally. www.rompa.com
Sullivan’s Heroes is a charity offering support and financial assistance for families raising essential funds towards vital home adaptations for a disabled child. Whilst council grants may be available, in many cases these don’t fulfil the required needs of a building project. www.sullivansheroes.org
Takeda are a global leader in clinical research into genetic therapies, and are currently involved in the investigations concerning several neurodegenerative conditions. www.takeda.com/en-gb
Thomas’s Fund is a charity that provides music therapy in Northamptonshire for children and young people with life-limiting illnesses or a disability which, for medical reason, means they are too ill to attend school for extended periods. www.thomassfund.org.uk
Together for Short Lives is the leading UK charity for all children with life-threatening and life-limiting conditions, and all those who support, love and care for them. Their work aims to ensure that children can get the best possible care, wherever and whenever they need it. www.togetherforshortlives.org.uk