AMN symptoms and their impact on men

About Raremark Raremark’s vision is to create a future in which rare medical conditions are as recognized as the common 05/12/2018

Patient network in rare disease We’re active in 13 therapeutic areas 05/12/2018

Data collection through surveys We run patient surveys on subjects including: Location Diagnosis Treatment Symptoms Quality of life 05/12/2018

Bridging the gap with industry History of diagnosis Diagnosed patients interested in new treatments Input to clinical trial design Real-world experience 05/12/2018

Insights in adrenoleukodystrophy (ALD)

ADHD the most common misdiagnosis 5 years On average, it took five years* for someone to be diagnosed from first experiencing symptoms 39% 39% of people received at least one misdiagnosis ADHD Most common misdiagnosis. Others included: fibromyalgia, neuropathy, anxiety and depression Sample size = 33 participants, surveyed March 2017. *Based on 29 participants who were diagnosed after experiencing symptoms. 05/12/2018

“Most doctors we dealt with for my grandsons had no knowledge of ALD/AMN so we usually educated them about the disease. My neurologist now knows more about ALD/AMN than any other doctor we have dealt with through the entire process.”

ALD Life/Raremark AMN survey

Where are the knowledge gaps? AMN survey Adrenomyeloneuropathy (AMN) is a rare inherited metabolic disorder that affects around one in 25,000 people Where are the knowledge gaps? 05/12/2018

Who’s the AMN survey for? Men aged 18 or over Confirmed diagnosis of AMN Living in the UK Have not previously had a bone marrow transplant Why take part? http://www.surveygizmo.com/s3/3910216/AMN-impact-Raremark-ALD-Life-research-project 05/12/2018

Get in touch: emily.armstrong@raremark.com pete.chan@raremark.com HONcode standard for trustworthy health information 05/12/2018 12 05/12/2018