We are delighted to have joined forces with Action Medical Research (AMR) and other Niemann Pick Charities (Niemann-Pick UK, NP Suisse and Niemann-Pick de Fuenlabrada) to support Professor Frances Platt conduct a project that starts this month and will run for 24 months. We have made a material contribution to this six-figure fund and are grateful to the AMR for their ongoing support of NPC research.
The Platt Lab are working with Professor Sarah Spiegel, at Virginia Commonwealth University in America, to repurpose an existing multiple sclerosis drug which they expect to boost the levels of the NPC1 protein and therefore be useful for treating patients with NPC. They will also test it in combination with an existing approved NPC drug, Zavesca (Miglustat), to see if the benefits are further increased.
This is a fantastic opportunity for the NPRF to join forces with other charities to fund bigger projects that would be financially challenging for us alone and make the funds you help us raise go further. We hope to repeat this model of funding as we continue to support research into this ultra rare disease.
For further information, please visit Action Medical Research’s page https://action.org.uk/research/niemann-pick-disease-type-c-testing-potential-new-drug-treatment