Since our inception we have went from strength to strength; we were granted charitable status as an independent charity in 1997, and in January 2012 (whilst remaining a registered charity), we changed our unincorporated charitable status to that of a charitable company limited by guarantee.
Over the last twenty years, we have seen major developments within the Niemann-Pick disease world, with advances in technology and increased knowledge and understanding bringing heightened interest and awareness to this group of diseases…which in turn has meant we can do more for the individuals affected by Niemann-Pick! However we do still rely solely on membership fees, voluntary donations, fundraising events and charitable grants for our income. Without this funding we wouldn’t be where we are today. In the video below, NPUK Founder & Former Trustee Jim Green explores both how NPUK came to be and how there are positive signs for the community going forward:
Below you’ll find just a few of our historic dates of which we are so proud…but here’s to hoping there will be many more to come in the future!
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