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#GoMakeMemories #ShortFilm #NPUK

Go Make Memories is the latest NPUK produced short film which centres on a young family and the impact of a Niemann-Pick diagnosis. It uses a mixture of both animation and live-action to better express the effects of this devastating disease on both the individual and the family - but also that there is positivity and hope amongst even the darkest moments...

Director/Producer: Carl Mason & Executive Producer: John Lee Taggart

Animation by James Richardson

Talented animator James Richardson handled the animated sections of the film, providing the expertise needed to take the project to the next level. Go Make Memories is different to any other awareness short you may have watched in the past, allowing for us to better display the progression of Niemann-Pick disease type C...

Go Make Memories to screen in San Francisco

We are pleased to announce that the NPUK produced short film Go Make Memories will feature in the DISORDER: Rare Disease Film Festival, in November 2019 (San Francisco) - after which we are hoping that the film enjoys a positive festival run in 2019/2020

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Latest news

19.11.19

Orphazyme receives Breakthrough Therapy Designation for arimoclomol in Niemann-Pick Disease Type C (NPC)

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.”

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31.10.19

Cyclo Therapeutics Announces Its Publication of the Most Extensive Set of Case Study Reports To-date on Expanded Access Use of Hydroxypropyl Beta Cyclodextrin in Niemann-Pick Disease Type C

Publication describes favorable safety profile and efficacy in neurologic and other disease features in individual NPC patients following intravenous administration of drug...

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