Research: Working with new partners

25th February 2015


We have been working closely with research institutions to help find better treatment for Adrenoleukodystrophy (ALD) and Adrenomyeloneuropathy (AMN). Funding for research into ALD and AMN is limited – that’s why research institutions play an important role in finding out more about the condition.

In 2013, we have been working as part of the ALD-AMN Global Alliance with Bluebird Bio. Bluebird Bio have been developing a Starbeam Study (ALD-102) as a potential to treat patients with Adrenoleukodystrophy. Asif Paker MD MPH held a lunch time presentation at our last Get Together Family Weekend looking at their study in detail.

More recently, we have been working closely with Minoryx, a pharmaceutical company in Spain – specialising in creating a small molecule drug class aimed to restore protein functionality. Minoryx secured 1.6 million euro funding last month to start a clinical trial for Adrenoleukodystrophy.

If you are a pharmaceutical company, and would like to work with us to support sufferers with adrenoleukodystrophy, please get in touch:
A 45 Peckham High Street, London, SE15 5EB
T 0207 701 2628


20 hours ago

ALD Life

Emory University is looking for families in Georgia with individuals who were diagnosed through newborn screening with ALD, as part of efforts to include ALD in the Georgia newborn screening panel. Participants would be speaking with Emory University via phone or email at their convenience. If you would like to give your input, please reach out to Dawn Laney with Emory University at or ‪404-778-8518‬. ...

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