Rare Diseases Related News
European Commission to launch knowledge-sharing platform for rare diseases
To support better diagnosis and treatment, the European Commission is launching a new online knowledge-sharing platform for more than 30 million Europeans living with...
Let’s delve into the world of rare diseases and the healthcare ecosystem
Today marks Rare Disease Day and it’s time Europe’s healthcare ecosystem adapts to embrace the potential of gene therapy for rare diseases.
28 February 2019...
Wilson Disease and the wonderful world of rare disorders
MedUni Vienna researchers have discovered a recently described genetic mutation could protect against fatty liver and possibly ensure a better outcome for Wilson disease.
Copper...
The custom-built molecule designed to battle a rare genetic disease
Scientists at the University of Bath, King's College London and Brunel University London, UK, have created a molecule offering protection to patients with rare...
Exploring mucopolysaccharide storage disorders
Susanne Gerit Kircher, Medical University of Vienna, argues that Europe must renew its efforts to tackle rare diseases such as mucopolysaccharide storage disorders.
Mucopolysaccharide storage...
The New Modalities Ecosystem: a much-needed boost for rare diseases
The collaborative New Modalities Ecosystem unites academia, pharma and technology companies to drive real and meaningful progress in research and treatments for rare diseases.
IN...
Patient groups: our strongest weapon against rare diseases
Findacure CEO Rick Thompson sets out the crucial role of patient groups in developing new treatments for rare diseases.
Rare diseases are defined in Europe...
Fit for purpose? The European Reference Networks, two years on
EURORDIS-Rare Disease Europe’s ERN and healthcare director, Inés Hernando, reflects on the progress of the European Reference Networks two years on.
Launched by the European...
Rare diseases in the Finnish landscape
Health Europa introduces the New Modalities Ecosystem, which will exploit Finnish expertise to deliver new treatments and technologies for rare diseases.
The New Modalities Ecosystem...
Therapies for recombination-deficient severe combined immunodeficiency
Leiden University Medical Center, Netherlands, is developing stem cell-based gene therapy for recombination-deficient severe combined immunodeficiency.
Leiden University Medical Center is the co-ordinating partner for...
Uterine sarcoma – tackling the rare aggressive cancer
Published in Clinical Cancer Research, new insights into uterine sarcoma, a rare type of aggressive cancer, opens up novel avenues of study.
Revealed by Karolinska...
Becoming reality – mucopolysaccharide storage diseases and adulthood
Susanne Gerit Kircher, of the Medical University of Vienna, explains why more resources and effort must be put into understanding mucopolysaccharide storage diseases in...
A call to action for idiopathic pulmonary fibrosis
The EU-IPFF sets out a series of recommendations to improve the quality of life and treatment outcomes of people living with idiopathic pulmonary fibrosis...
Rare blood disorders: could sutimlimab treat the untreatable?
Researchers yearn to treat rare blood disorders, and now they may have found the solution with the investigational drug, sutimlimab.
Sutimlimab has shown promising results...
Nosocomial infections: the potential killer Staphylococcus epidermidis
Staphylococcus epidermidis is a pervasive coloniser of healthy human skin, but is also a notorious source of serious nosocomial infections found in hospital devices.
Scientists...
Study reveals how aid communication is perceived in African countries
Aid recipients call for more dignity and diversity in aid communication from international NGOs (INGOs) and development organisations.
Participants from six Sub-Saharan African countries spoke...
Gene therapy process reversing sickle cell anaemia symptoms
After over a decade of preclinical research and development, a new gene therapy process is reversing sickle cell anaemia symptoms in patients.
The gene therapy...
Rotavirus infection influenced by complex interactions in mother’s milk
Researchers have found that the sugars and microbiome in mother's milk influence neonatal rotavirus infection.
An international team of researchers from several institutions, used a...
Rare forms of cancer to be tackled by Asian-European collaboration
ESMO launches new partnership to boost Asian-European collaboration and tackle rare forms of cancer, with the launch of Rare Cancers Asia.
The European Society for...
Maybe the princess kissed the frog because of ranavirus?
New research has shown frogs from groups that are exposed to the deadly disease, ranavirus, breed at younger ages to beat the epidemic.
Scientists from...