News

Researchers say that adding a common altitude sickness drug to the treatment protocol for patients with glioblastoma may improve the effectiveness of chemotherapy and extend survival in some patients.

The first human clinical trial to assess RNA therapy, QR-313, for the treatment of recessive dystrophic epidermolysis bullosa has been initiated.

Elagolix (Orlissa) is the first oral gonadotropin-releasing hormone antagonist specifically developed for women with moderate to severe endometriosis pain.

A study of pediatric patients with asthma admitted to emergency departments has determined that while rhinovirus is the most prevalent infection, it is the non-rhinovirus pathogens that are associated with an increased absolute risk of treatment failure.

Researchers found that just 18.4% of young women diagnosed at pediatric emergency departments with pelvic inflammatory disease received HIV and syphilis screening, despite their higher risk for those sexually transmitted infections.

A workgroup convened by the Alzheimer’s Association has developed a list of 20 recommendations regarding symptoms of Alzheimer’s disease and related forms of dementia.

Researchers find that a glucose-derived dietary supplement was able to increase muscle-force production in a Duchenne muscular dystrophy mouse model by 50% in 10 days.

PharmaKrysto Ltd. and Rutgers University, New Jersey, have entered into a licensing agreement for the development of PK10, a novel drug for the treatment of cystinuria.

The PocketECG Cardiac Rehabilitation System provides electrocardiography (ECG) monitoring and alerts during cardiac rehabilitation.

Researchers found that enhanced disinfection with UV light in targeted rooms led to a decrease in hospital-wide incidence of C. difficile and vancomycin-resistant enterococci.

A review of literature on depression and anxiety in patients with heart failure reports that 21.5% have depressive symptoms and 19% meet the criteria for a depressive disorder—2 to 3 times higher than rates in the general population.

Stay up-to-date on the latest rare disease news by reading the top 5 articles of the week.

Researchers have discovered that a gene may be linked to neuromyelitis optica, a highly rare disease known to lead to blindness and paralysis.

The FDA has approved ivosidenib tablets for the treatment of adult patients with relapsed or refractory acute myeloid leukemia who have a specific genetic mutation.

The US Food and Drug Administration (FDA) has granted an orphan drug designation to Corbus Pharmaceuticals Holdings, Inc’s lenabasum for the treatment of dermatomyositis.

One phase 2 study showed that RBX2660, a microbiota-based drug comprised of human stool, was effective in 77.8% of patients.

The US Food and Drug Administration (FDA) has granted fast track designation to galinpepimut-S (GPS) for the treatment of multiple myeloma.

The cerebrospinal fluid assays can detect biomarkers of Alzheimer disease and help clinicians diagnose AD before clinical symptoms appear.

Proper diagnostics is key in healthcare, but patient education on management and medication use can lead to empowerment that unlocks optimal health outcomes.

The ACAAI's new yardstick for pediatric asthma provides guidance on stepping-up asthma treatments to gain and maintain symptom control.

The statistical algorithm incorporates data including age, depression severity, neuroticism, and cognitive control to decide which patients with depression are most likely to benefit from the antidepressant sertraline.

New findings suggest that a specific hemoglobin production protein could be a target for therapy in sickle cell disease or beta-thalassemia.

The FDA has granted a breakthrough therapy for atezolizumab Tin combination with bevacizumab for the treatment of advanced or metastatic hepatocellular carcinoma.

Perhaps surprisingly, patients admitted during periods of higher occupancy had 15% lower odds of contracting Clostridium difficile infection.

Researchers have identified a unique mutation in a deceased transthyretin (TTR) amyloidosis patient, which sheds light on the disease and may help identify new targets for treatment.

Researchers found that tobacco users, regardless of what product-type they used, underestimated the risks nicotine exposure posed to children and the high rates of multiple tobacco use.

The first meta-analysis of AMD clinical meta-analyses shows many have methodological limitations—more so those sponsored by industry than those sponsored by governments.

The FDA has granted an orphan drug designation to Asceneuron SA for ASN120290, for the treatment of progressive supranuclear palsy.