Dr Julie Vallortigara discusses insights from the report on the state of patient-centricity in clinical trials and the role patients play at the charity. 

As part of the Partnerships in Clinical Trials conference edition 2017, Dr Julie Vallortigara, our Research Officer, had the opportunity to share her views and comments on a report recently published by KNect365 Life Sciences on the state of patient-centricity in clinical trials. You can read the report here and the article by Julie Vallortigara can be found here. Dr Vallortigara is speaking on ‘Working with patient groups for clinical research’ and ‘How to attract funding for rare diseases’ on 29 November at Partnerships in Clinical Trials Europe in Amsterdam.

Posted: 15/11/2017

Agilis announces orphan product designation approval in Europe for the treatment of Friedreich's ataxia

This is the first gene therapy treatment candidate to receive orphan designation in EU and USA. Agilis Biotherapeutics, Inc. (Agilis), a biotechnology company advancing innovative DNA therapeutics for rare genetic diseases that affect the central nervous system, announced on 31st October that the European Commission (EC) has granted Orphan Medicinal Product designation in the European Union to the Company’s gene therapy product candidate, AGIL-FA, being developed for the treatment of Friedreich ataxia. The EC’s approval follows a positive opinion in July 2017 from the European Medicine Agency’s Committee for Orphan Medicinal Products. This follows the Orphan Drug Designation for AGIL-FA granted by the U.S. Food and Drug Administration last year. For more information, read the press release.

Posted: 07/11/2017

Biohaven reports negative topline data from spinocerebellar ataxia (SCA) phase 2-3

Posted: 07/11/2017

BioMarin announced one compound in the pipeline for the treatment of Friedreich's ataxia 

BioMarin announced that it has selected BMN 290, a selective chromatin modulation therapy, for the treatment of Friedreich's Ataxia (FA). In preclinical models, BMN 290 increases frataxin expression in affected tissues more than two-fold.  BMN 290 is a second generation compound derived from a compound acquired from Repligen that had human clinical data demonstrating increases in frataxin in FA patients.  BMN 290 was selected for its favorable penetration into the central nervous system and cardiac target tissues, and its preservation of the selectivity of the original Repligen compound. You can read the press release here. Ataxia UK has met with Biomarin team in London and will be discussing future trial using this new compound once the company is able to run the study.

Posted: 07/11/2017

IARC 2017 highlights! 

The International Ataxia Research Conference happened last week in Pisa! This event was very well attended and great research was reported there as well as patients personal stories and need for treatment. We will be blogging about this conference very soon and will report back in our magazine. In the meantime you can read some blogs done by Friedreichsaataxianews.

Posted: 04/10/2017