All our efforts and funds are used for three main range of activities:
SUPPORTING PATIENTS AND FAMILIES
We are a patients’ support organisation and our core activity is providing support for patients and families with a diagnosis of ALD or AMN in their journey through the conditions. Patients with a diagnosis of a rare disease are not provided with sufficient reliable information on their medical, psychological, social and other needs. Having a diagnosis of rare disease is very easy to feel alone and isolated. No one understands your disease, often neither do your GP or your specialist.
We provide them with practical information, contact with others in similar circumstances, emotional and financial support through our grant programme for funding Bone Marrow Transplantation and bereavement.
It is a sad fact of ALD that there must be a place for bereavement on this website. If you have suffered the loss of a loved one and would like to be put in touch with someone who has experienced loss this before, please contact us here.
AWARENESS RAISING
As we work with rare genetic diseases, improving the awareness about ALD (adrenoleukodystrophy) and AMN (adrenomyeloneuropathy) is fundamental to us. It contributes to make the patients and their families feeling less isolated.
We promote campaigns and information to raise the awareness among the general public and, especially, among the NHS professionals. We promote training for specialists and we attend conferences and workshops, at national and international level, to speak loud about the needs of our patients and their families, the special needs of patients with rare diseases, make the professionals aware about ALD and AMN with the final aim of improving the quality of care for our patients.
RESEARCH
The research for rare diseases is unfortunately very poor and there is limited funding provision for research project around new treatments for ALD and AMN.
As Rare disease UK report in one of their recent publication “the amount of funding provided for research into rare conditions in the UK is limited. Although it is not possible to obtain exact figures of the total funding for rare disease research, the Association of Medical Research Charities (AMRC) estimates that in 2008/09 charities invested approximately £3.6 million in rare disease research, compared to £370 million in cancer research. Based on best estimate figures suggesting that 3.5 million people are affected by rare diseases, this equates to £1 of charitable funding per patient, compared to £185 per patient for cancer (based on 2 million affected people)”
Without a systematic programme of research, progress in the development of diagnostic tests and treatments for rare conditions is greatly hindered.
That is why we have decided to raise funds for supporting scientific research all over the world for finding new treatments and a cure for ALD and AMN.