New app helping Fabry’s disease patients


Royal free

New app helping Fabry’s disease patients

Patients with a debilitating rare condition have had their lives transformed thanks to a new app at Royal Free Hospital which allows doctors to monitor symptoms remotely and adjust their treatment accordingly. Since the app was launched last July about


Treatment of thoracolumbar kyphosis in patients with MPS I

Patients with mucopolysaccharidosis type I (MPS I), skeletal disease (dysostosis multiplex) is a prominent, debilitating, condition related complication that may impact strongly on activities of daily living. Unfortunately, it is not treated with hematopoietic cell transplantation (HCT) or enzyme replacement


Finding love or just find love for yourself. Either way, let’s help— whether you have a disability or not.

Having a disability changes your life and will continue to do so but with greater self-confidence and a positive self-image, comes the ability to face challenges, adapt, and evolve. When you focus on your flaws, you are telling others to

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News from WORLDSymposium 2019

All the news from the MPS Society attending WORLDSymposium 2019 4-8 February. WORLDSymposium™ is an annual research conference dedicated to lysosomal diseases. W.O.R.L.D. is an acronym that stands for We’re Organizing Research on Lysosomal Diseases. Since its inception as a small group of


Poster presentation at WORLDSymposium, February 2019, Orlando, Florida, USA

WORLDSymposium in Orlando where leading organisations get together to discover latest discoveries in lysosomal diseases. This year, over 440 abstracts will be presented at the two WORLDSymposium poster sessions. These sessions are an excellent opportunity to see, hear and discuss specific research