NPUK is a member of the UK LSD Patient Collaborative Group. The collaborative is comprised of patient organisations representing those affected by lysosomal storage disorders (LSDs) who have joined forces to create a strong lobbying and action group for LSD patients and their families in the UK.
The collaborative aims to provide an understanding of LSDs by working in partnership to advance standards of care and to enhance the wellbeing of those affected by LSDs. See below for a list of the members and to find out more go to lsdcollaborative.org.uk.
LSD UK Patient Organisation Secretariat
8 Silver Street
Gloucestershire GL11 4ND
Tel: 00 44 1453 549231
Fax: 00 44 1453 549231
The AGSD provides support and help for individuals and families affected by Glycogen Storage Disease (GSD).
The Batten Disease Family Association (BDFA) is a national charity which aims to support families, raise awareness and facilitate research into the group of devastating neurodegenerative diseases commonly known as Batten Disease.
The CATS Foundation supports families affected by Tay-Sachs by offering equipment, information, and funding for research.
The Gauchers Association promotes awareness, pioneers research and provides support and information for those affected by Gaucher disease.
The Society for Mucopolysaccharide Diseases (The MPS Society) is a voluntary support group founded in 1982, which represents from throughout the UK over 1200 children and adults suffering from Mucopolysaccharide and Related Lysosomal Diseases including Fabry Disease, their families, carers and professionals.
We, NPUK, are a charitable organisation dedicated to making a positive difference to the lives of those affected by Niemann-Pick diseases. We raise much needed awareness, provide practical and emotional support, advice and information and facilitate research into potential therapies.
Kim Stratton, Chief Executive Officer at Orphazyme, commented, “We are delighted with FDA’s decision to grant Breakthrough Therapy Designation to arimoclomol for NPC. Arimoclomol has been shown to have a clinically meaningful effect on disease progression in NPC that is further supported by a biomarker effect indicating an effect on the biological underpinnings of the disease and a favorable safety and tolerability profile. We are committed to bringing this product to patients as soon as possible. Breakthrough Therapy Designation is designed to expedite the development and review of products for serious diseases with the direct involvement of senior staff and we look forward to working closely with the FDA to further advance arimoclomol. Our preparations for filing in the US are underway and we are on track to submit a New Drug Application in H1 2020.”Read more