The 1983 US Orphan Drug Act resulted in a significant and sustained increase in treatments for rare diseases. The thousands of rare genetic diseases affect over 10 million Americans. A rare disease is defined as a disorder with a prevalence of less than 200,000 in the United States [1]. Thousands of drugs are being developed for rare diseases; however, the majority of treatments are for rare on...

Tuberculosis (TB) remains a significant cause of death worldwide, and emergence of drug-resistant TB requires lengthy treatments with toxic drugs that are less effective than their first-line equivalents. New treatments are urgently needed. Delamanid, previously OPC-67863, is a novel drug of the dihydro-nitroimidazole class with potent anti-TB activity and great promise to be effective in the t...

Millions of people and animals suffer from superficial infections caused by a group of highly specialized filamentous fungi, the dermatophytes, which only infect keratinized structures. With the appearance of AIDS, the incidence of dermatophytosis has increased. Current drug therapy used for these infections is often toxic, long-term, and expensive and has limited effectiveness; therefore, the ...

Both surgical myectomy and percutaneous transluminal septal myocardial ablation are effective treatments for drug-refractory symptomatic hypertrophic obstructive cardiomyopathy (HOCM). However, in some cases, it is not easy to elucidate the abnormal structure of left ventricular outflow obstruction to adopt these treatments. Here, we presented a young female patient with drug-refractory symptom...

Extensive evidence from the laboratory and the clinic suggests that drug addiction can be viewed as operant behavior and effectively treated through the application of principles of operant conditioning. Contingency management interventions that arrange for the direct reinforcement of drug abstinence or of other therapeutically important target behaviors (e.g., regular use of drug abuse treatme...

The ability to computationally predict whether a compound treats a disease would improve the economy and success rate of drug approval. This study describes Project Rephetio to systematically model drug efficacy based on 755 existing treatments. First, we constructed Hetionet (neo4j.het.io), an integrative network encoding knowledge from millions of biomedical studies. Hetionet v1.0 consists of...

Antibiotic-resistant bacteria pose a serious threat to immuno-compromised individuals in Intensive Care Units (ICU). This study examines several cycling treatments (7,14,30,60,120,240-day cycle) and random fraction treatment (50-50,60-40,80-20,100-0) strategies in ICU and finds that no single strategy will outperform all others. Human, hospital and pathogen conditions such as admission/departur...

A compartment model for the evolution of cancer subject to multiple chemotherapeutic agents is presented. The formulation accounts for the heterogeneous nature of cancer and drug resistance, leading to a system that can be used as a tool for optimizing treatments. Chemotherapeutic or cytotoxic agents have limited effectiveness due to of toxicity and the development of drug resistance. Toxicity ...

Our aim was to review and compare findings from controlled trials and previous reviews concerning current drug treatment of patients suffering from bulimia nervosa (BN) and binge eating disorder (BED). Thus we selected published articles quoted over the last 10 years in the databases of Medline and Cochrane Library. The combination of pharmacological and psychological treatments is superior to ...