Isabelle Richard's team, a CNRS researcher in an Inserm unit at Genethon, the AFM-Telethon laboratory, has demonstrated the efficacy of gene therapy and determined the effective dose for treating a rare muscle disease, gamma-sarcoglycanopathy, in mouse models of the disease. Based on these encouraging results, published in Molecular Therapy: Methods and Clinical Development, the researchers are preparing a clinical trial.
Research led by a UCLA scientist found that a new nerve stimulation therapy to increase blood flow could help patients with the most common type of stroke up to 24 hours after onset.
Parent experiences when approached for research in a pediatric intensive care unit, the role of inclusion benefits in ethics committee assessment of research, and more in the current issue.
A new treatment approach that involves blocking a protein has, for the first time, shown promising results in the treatment of fatty liver disease, in a landmark translational research collaboration between Duke-NUS Medical School, National Heart Centre Singapore and biotech company Enleofen Bio.
At ASCO 2019, SWOG investigators will report on 16 group-led trials, one jointly-led study, and another 12 involving partners. Presentation topics illustrate SWOG's wide-ranging portfolio, with talks and posters on treatment or prevention of bladder, breast, colorectal, lung, pancreatic, prostate, and rare cancers, as well as melanoma and multiple myeloma. SWOG investigators will make a particularly strong showing in symptom control, survivorship, and quality of care trials.
A new study from Children's Hospital of Philadelphia finds that a skin patch may be useful in treating children with a painful, chronic condition called eosinophilic esophagitis (EoE) triggered by milk. Among 20 children with EoE who wore Viaskin Milk -- a skin patch measuring just over an inch long containing trace amounts of milk protein -- nine saw an improvement in their symptoms and normalization of their biopsies after 11 months.
People who suffer a stroke caused by bleeding in the brain -- known as brain haemorrhage -- can take common medicines without raising their risk of another stroke, a major clinical trial has found. Researchers say the findings are reassuring for the thousands of people who take the medicines to reduce their risk of heart attack and another common type of stroke caused by blood clots in the brain.
An analysis of 117 cancer drugs approved by the US FDA over a 20-year period finds the drugs took a median of 6.5 years to go from the first clinical trial in adults to the first trial in children. 'As a doctor taking care of young cancer patients, this is tremendously frustrating,' says lead researcher Steven DuBois, M.D. 'If I were a parent of a child with cancer, I wouldn't stand for this.'
An anterior cruciate ligament (ACL) tear, an injury of the knee, can be devastating to a young athlete. While the ACL can be reconstructed through surgery, there is a high risk of re-injury in patients under the age of 25. In the largest clinical trial of its kind, researchers at Lawson Health Research Institute have shown that performing an additional surgical procedure called lateral extra-articular tenodesis (LET) may reduce the risk of ACL re-injury in young athletes.
'This study shows that [rare pulmonary side effects of brigatinib] can disappear within days despite continued exposure to the drug,' says D. Ross Camidge, M.D., Ph.D.