Please login to the form below

Not currently logged in
Email:
Password:

Digital intelligence blog

Pharma insight on digital marketing, social media, mobile apps, online video, websites and interactive healthcare tools

PatientsLikeMe takes aim at one of world's rarest diseases

Creates open registry and community for alkaptonuria patients

PatientsLikeMe alkaptonuria AKU communityOnline patient community PatientsLikeMe has expanded its networking efforts to one of the world's rarest diseases, setting up the world's first open registry for patients suffering from alkaptonuria (AKU).

The first genetic disease to be discovered, AKU is estimated to affect one person in every 250,000-500,000 and leads to a condition that causes the bones and cartilage to become black and brittle.

Sufferers will now be able to track their disease progression through PatientsLikeMe, connect with others who have the disease and contribute health data to the registry's real-time research platform.

PatientsLikeMe co-founder and chairman Jamie Heywood said: “This open registry will give anyone—hospitals, pharmacies, providers, nonprofits and patients themselves—a real world view of where and how the disease affects people.

“This information is vital to deepen our collective understanding of AKU and to drive smarter action, more effective treatments and better patient outcomes.”

AKU Society Chairman Nick Sireau said the registry could shed important new light on the disease.

“More than 100 years after its discovery, we still don't know exactly how many people have AKU, or what they are doing and experiencing. We're excited to partner with PatientsLikeMe to help patients connect with each other and help researchers answer some of the most fundamental questions about rare diseases.”

PatientsLikeMe will work with the AKU Society to gather data about AKU patients' symptoms and daily lives to establish the most up-to-date source for new medical evidence about the disease.

Although there is currently no cure for AKU, Swedish Orphan Biovitrum's tyrosinaemia treatment Orfadin (nitisinone) is used off-label to treat AKU and is expected to enter phase III trials for the condition later this year.

14th January 2013

From: Healthcare

Share

Tags

COVID-19 Updates and Daily News

Featured jobs

PMHub

Add my company
Fishawack Group of Companies

The Fishawack Group of Companies is one of the largest independent medical communications and medical marketing specialists, with teams in...

Latest intelligence

What challenges still face clinical trial recruitment and retention?
Discover the reasons for the current clinical trial recruitment challenges facing pharma and the life sciences industry, and how the search for solutions to the problem continues...
“Fake News” and Credibility in Medical Publishing
Natalie Yeadon from Impetus Digital shares her thoughts on fighting fake news in Pharma and healthcare....
Delivering true value: what does it mean for KAM in cancer care?
Lisa Alderson, Business Development Director at Wilmington Healthcare, explores the challenges that pharma’s KAM teams face in engaging with the NHS and how they must evolve...

Infographics