Regulators

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Name

Sobi

Location

Stockholm, Sweden

Contact

Mattias Rudebeck

Website

http://www.sobi.com/ 

Description

Sobi is a Swedish based leading integrated biopharmaceutical company dedicated to bringing innovative therapies and services to improve the lives of rare disease patients and their families. Sobi currently markets nitisinone (under brand name: Orfadin®) for use in patients diagnosed with hereditary tyrosinaemia type I.

Sobi supplies nitisinone to DevelopAKUre, and also provides regulatory and pharmacovigilance support and will submit the final data following a positive program outcome to the European Medicines Agency for potential approval of nitisinone as a treatment for AKU.


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Name

PSR Group

Location

Hoofddorp, the Netherlands

Contact

Christa van Kan

Website

http://www.orphandrugexperts.com/site/home/

Description

PSR is a leading orphan drug development CRO (contract research organisation), which specialises in the design and implementation of clinical trials in rare diseases.

PSR is the medical monitor, and trial manager for DevelopAKUre, ensuring all clinical sites follow the protocols and process and store data correctly. PSR is also responsible for submission of ethical and legal documents.


 

 
 

Welcome To DevelopAKUre


DevelopAKUre is a series of major international clinical trials, run by a consortium of 12 European partners. It aims to study a potential new drug, called nitisinone, and assess its potential effectiveness in treating the rare disease, alkaptonuria (AKU).

DevelopAKUre is co-funded by a grant from the European Commission. This website is run by a UK patient group, the AKU Society. Learn more about AKU on the AKU Society's What is AKU page.

 

DevelopAKUre Blog

SONIA 2 – Controlling Protein to Support Nitisinone Treatment
11-Jul-2017

In today’s blog Chief investigator Prof. Ranganath reinforces the need for SONIA 2 pat.. ...more

Introducing PhD Student Brendan Norman
16-Feb-2017

In this week's blog, we talk to Brendan Norman, a PhD student at the University Of Liverpool, ab.. ...more