You’ll find an extensive list of organisations and services over at the ‘Useful Resources and Links’ section of this site…as the name suggests they can be rather handy, and may be of great assistance to you!
But please remember: we want this site to be a place where information is easily accessible, and also understandable – so if that is ever not the case, don’t hesitate to get in touch with us so that we can talk you through anything complicated. We will do everything in our power to help you.
We are always just a phone call (0191 415 0693) or email (firstname.lastname@example.org) away. So you needn’t ever feel lost or disillusioned as you always have a friend in NPUK.
(Keeping a record of the people who help you can make it easier to get support when you need it. Try to keep a list of their contact details handy – or get in touch with us and we will provide you with all we have!)
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