London, UK – Patients with rare diseases could benefit from a “revolution” in clinical trials that could see one-stop studies designed that provide robust results even with small numbers of participants. The CAPTIVATE node is part of the recently introduced UK Rare Disease Research Platform established as part of a...
research News
DURHAM, N.C. — Children born without thymus glands have given Duke University Medical Center researchers a rare opportunity to watch as a new immune system develops its population of infection-fighting T-cells. Researchers led by Thomas Kepler, Ph.D., Division Chief of Computational Biology, tracked three young patients after thymus tissue transplantation...
London, UK – Research led by Claudia Cooper, Professor of Psychological Medicine at Queen Mary University of London, shows that a new therapy, NIDUS-Family, helps people with dementia and their family carers attain their personal goals. The NIDUS-family package of care and support focuses on practical changes people can make, with sessions designed around the specific priorities of the...
QUT researchers have signed an agreement with US pharmaceutical company Quoin to fund a pre-clinical program at QUT to further research a potential treatment for a rare skin condition called Netherton Syndrome. Research will focus on Netherton Syndrome (NS), an hereditary autoimmune condition NS is characterised by skin inflammation, itchiness,...
Montreal, Canada – In Quebec, it is estimated that more than 500,000 people suffer from a rare disease, which is defined as a disease that affects less than one in every 2,000 citizens. Too often, affected patients and their families are left in the dark when it comes to finding...
Dublin, Ireland – Researchers in the School of Population Health at RCSI University of Medicine and Health Sciences have provided new evidence of the health benefits of weight loss efforts that lead to diabetes remission for type 2 diabetes patients. For participants in the weight-loss trial who were able to...
Dublin, Ireland. Boosting levels of a deficient protein has clear survival benefits for people with the genetic condition alpha-1 antitrypsin deficiency (AATD), according to new research led by RCSI University of Medicine and Health Sciences. The findings underscore a call to make augmentation therapy accessible to patients with AATD in...
Dublin, Ireland – A new partnership between RCSI University of Medicine and Health Sciences and Irish diagnostics company Serosep Ltd is set to personalise treatment for ulcerative colitis and ultimately improve quality of life for patients with the condition. Under the new alliance, RCSI researchers will work with Serosep to...
LA JOLLA, CA — Nerve cells in the brain demand an enormous amount of energy to survive and maintain their connections for communicating with other nerve cells. In Alzheimer’s disease, the ability to make energy is compromised, and the connections between nerve cells (called synapses) eventually come apart and wither,...
Amsterdam, Netherlands – Findings from a new study in the Journal of Neuromuscular Diseases, published by IOS Press, demonstrate the effectiveness of disease-modifying treatments (DMTs) in infants with spinal muscular atrophy (SMA). The study’s results add further support for gene therapy as a treatment modality that can deliver durable transformative...