News

FDA approves Phase 2 study of KD025, a a possible treatment for graft-versus-host disease (GVHD).

While some baseline characteristics may put patients at a greater risk of developing macular atrophy, ultimately, ranibizumab was not determined to raise that risk.

Researchers can agree that medical errors are a big problem in the US. But can they agree on its pathology?

Drug resistance is increasing among HIV-positive patients, driven partly by transmission of resistance to and by ARV-naïve patients.

Jersey Mike’s Raises $377,000 for Make-A-Wish New Jersey.

CEO of Krystal Biotech discusses first topical gene therapy for dystrophic epidermolysis bullosa.

While conventional methods require some type of skin prick, scientists from the University of Bath have proven the concept of needleless CGM.

The findings indicate the potential for a clinically meaningful improvement and stabilization of lung function.

The device was able to accurately identify the presence, or lack thereof, of more-than-mild DR at least 87% of the time.

Results from a study published in Nature Communications offer potential treatment targets for melorheostosis, often referred to as “dripping candle wax” bone disease.

The self-administration of pnf C1-INH (Berinert) proves effective for patients with hereditary angioedema with C1 inhibitor deficiency.

Data from the article, “Fabry disease in the Spanish population: oberservational study with detection of 77 patients,” suggests that enzymatic screening for Fabry disease in risk populations assisted in identifying undiagnosed patients.

Investigators noted promising results for secondary outcomes but demonstrated no difference for the primary outcome of reducing hospital visits due to self-harm.

It is the first pharmacologic therapy approved in the United States for TSC, a rare genetic disorder that impacts roughly 50,000 patients in the US.

This morning, it was announced that everolimus (Afinitor DISPERZ) has become the first therapy to be specifically approved by the U.S. FDA to treat patients aged 2 years and older with tuberous sclerosis complex (TSC)-associated partial-onset seizures.

The team found that the children with both conditions had 2.2 times the risk of anxiety disorder and 2.7 times the risk of other mood disorders.

Long-term care facility residents with advanced dementia have substantial dysbiosis of their gut microbiome which may be able to identify subjects at high risk of C. difficile colonization.

Could cytotoxicity be conversely related to proinflammatory response in C. difficile cases?

Higher-quality epidemiological data is needed in all 4 subtypes of Sanfilippo syndrome for better disease research and management.

André the Giant, examines the life and work history of the WWE Hall of Famer. Unbeknownst to many, his size was the result of gigantism, a disease caused by excess growth hormone. The condition led to his eventual diagnosis with acromegaly, a rare endocrine disorder.

The contact lenses, which darken when exposed to strong UV light, were cleared through the 501(k) pathway.

In a recent interview with Dr Steven Freedman, Rare Disease Report asked for the specifics on familial chlyomicronemia syndrome (FCS).

Women with PCOS face increased risk of psychiatric morbidity and their children are more likely to be diagnosed with ADHD or autism.

The antipsychotic was approved by the FDA in 2016 on the backing of what researchers now consider unsubstantial clinical evidence.

The Global Burden of Disease study found that US mortality has improved since 1990 — but has been caused by worse health measures.

A new provisional patent was filed by Nutra Pharma Corporation to protect the intellectual property surrounding their development of a drug intended to treat amyotrophic lateral sclerosis.

The Prescription Drug User Fee Act goal date, which was originally set for Q2 of 2018 after the therapy was granted Priority Review designation, has now been pushed 3 months, to Q3.

Early this morning, Reviva Therapeutics announced that the U.S. Food and Drug Administration has granted orphan drug designation to its clinical stage drug candidate RP5063, in development for the treatment of idiopathic pulmonary fibrosis.

A higher prevalence of HCV in adults who received surgery for congenital heart disease as children is a "double whammy" when procedures leaves systemic venous congestion affecting hepatic veins, researchers said.

This is the first study to present nationally representative estimates of HIV testing prevalence and factors associated with HIV testing among sexually active older adults.