five steps from black bone disease...five steps from black bone disease dr nicolas sireau chairman...
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![Page 1: Five steps from Black Bone Disease...Five steps from Black Bone Disease Dr Nicolas Sireau Chairman and CEO, AKU Society Chairman and Co-founder, Findacure Step 1: Working with scientists,](https://reader036.vdocuments.us/reader036/viewer/2022071417/6115123d00325276291db9fa/html5/thumbnails/1.jpg)
Five steps from Black Bone Disease
Dr Nicolas Sireau Chairman and CEO, AKU Society
Chairman and Co-founder, Findacure
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Step 1: Working with scientists, clinicians and
industry to treat the disease
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The AKU tetrad
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AKU mouse model
Springer-Verlag
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Urinary HGA
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Step 2: Setting up a centre of excellence and
clinical trials
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The Robert Gregory National Alkaptonuria Centre
Funded by NHS England
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Trial Name Description Sites
SONIA 1: Suitability of Nitisinone in Alkaptonuria 1 3-month phase II study UK/Slovakia
SONIA 2: Suitability of Nitisinone in Alkaptonuria 2 4-year phase III UK/Slovakia/France
SOFIA: Subclinical Ochronosis Features
in Alkaptonuria Cross-sectional study UK
Three Studies
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The DevelopAKUre partners
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Step 3: Working with patients
around the world
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A global patient movement
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AKU Societies in EU, Asia, Middle East and North America • AKU Society UK
• ALCAP (France) • AIMAKU (Italy)
• AKU Society Germany • AKU Society Netherlands
• AKU Society Jordan • AKU Society India
• AKU Society Slovakia • AKU Society North America (USA and Canada)
• AKU Society Belgium (in progress)
• AKU Society Sweden (in progress) • AKU Society Asia (in progress)
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h"p://igg.me/at/curebbd
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Step 4:
Developing new therapies
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Step 5:
Sharing with other patient groups
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www.findacure.org.uk
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Rare Diseases: Challenges and Opportuni<es for Social Entrepreneurs
Out now! With chapters from leaders in the rare disease sector
Contact: [email protected]
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Presented by Genetic Disorders UK in partnership with Global Genes – Allies in Rare Disease
#GDLS2015