Rare Diseases Related News
bluebird bio announces launch of beta thalassaemia gene therapy in Germany
Zynteglo is a one-time gene therapy that addresses the underlying genetic cause of TDT and offers patients the potential to become transfusion independent.
The one-time...
MPS Austria: champions of self-help support and research for therapy
Mucopolysaccharidoses (MPS) are slowly progressive and inheritable metabolic diseases and MPS Austria helps to improve quality of life for MPS patients.
MPS diseases occur in...
Individual case of rare monkeypox disease confirmed in the UK
Monkeypox is a rare viral infection that does not spread easily between people.
Public Health England (PHE) have confirmed an individual case of the rare...
Health Europa Quarterly Issue 11
Health policy is in constant flux with healthcare models of every conceivable format facing growing pressure, here Health Europa Quarterly Issue 11 explores more.
Patients...
Everything you need to know about the annual MPS therapy week
From 11 July to 20 July 2019, the annual MPS therapy week and bi-annual MPS Conference was hosted in Hinterglemm, in the South-East of...
UK Tick-borne Lyme disease cases may be higher than thought
New cases of tick-borne Lyme disease in the UK may be three times higher than previous estimates suggest. The finding suggest that new cases...
Breakthrough monkey malaria study offers cure for relapsing malaria
A breakthrough in monkey malaria research by University of Otago, New Zealand, could help scientists diagnose and treat a form of relapsing malaria.
Published in...
Health Europa Quarterly Issue 10
Editor Sophie Clutterbuck introduces Health Europa Quarterly Issue 10, here our book delves into the world of Digital & E-health, medical cannabis, along with...
Light and shadow – daily life with Mucopolysaccharide storage disorders
Susanne Kircher, co-founder of the Austrian MPS Society, talks about the rare chronic diseases, mucopolysaccharide storage disorders.
Mucopolysaccharide storage disorders (MPS) are rare chronic diseases,...
Undiagnosed: are rheumatic and musculoskeletal diseases burdening the European taxpayer?
EULAR President Professor Johannes Bijlsma gives us an insight into the world of rheumatic and musculoskeletal diseases and the potential burden it could bring...
Did you know that using e-cigarettes appear to damage mucus clearance?
Published in the American Thoracic Society's American Journal of Respiratory and Critical Care Medicine, research shows that using e-cigarettes appears to impair mucus clearance.
According...
Promising clinical research could revolutionise mucopolysaccharidosis treatment
According to research, mucopolysaccharidosis treatment is still emerging, but recent drug approval & promising clinical research studies could accelerate treatment pattern for those suffering.
Mucopolysaccharidosis...
Universal health coverage – Smarter research & development to tackle global health priorities
WHO’s new Science Division launched an online resource to guide the development of Universal health coverage to further help combat global health priorities.
An essential...
Living with Transfusion-Dependent β-Thalassaemia (TDT)
In its most severe form and without adequate care, thalassaemia can be lethal in early childhood, and although Annual International Thalassaemia Day has come...
Non-melanoma skin cancer: The hidden epidemic faced by outdoor workers
We talk to Professor Swen Malte John, Chair of the EADV Task Force on occupational skin disease, who emphasises the magnitude of the neglected...
Discover the promising new Noonan syndrome treatment
According to researchers at Université de Montréal and CHU Sainte-Justine Research Center, Canada, there may be a new Noonan syndrome treatment on the horizon.
Noonan...
Have investigators discovered what triggers autoimmune liver diseases?
Discover the UK disease clustering study that suggests environmental exposure may play a key role in the development of some autoimmune liver diseases.
11 April...
A new innovation for gene editing may improve disease treatment
Purdue University, USA, have developed a new technology that could change how gene editing is approached and even improve disease treatment for the future.
Gene...
Canine Influenza virus: Is your pup spreading diseases?
Ever heard of Canine Influenza virus? Well, according to researchers, results from a 10-year study suggest two strains of influenza could mix to create...
The guide to tackling common zoonotic diseases
What do you know about common zoonotic diseases? Such diseases continue to be a threat to global health, but FAO, OIE and WHO may...