
: In a recent phase 3 SONICS study, levoketoconazole was found to be well tolerated in patients with endogenous Cushing’s syndrome.

: In a recent phase 3 SONICS study, levoketoconazole was found to be well tolerated in patients with endogenous Cushing’s syndrome.

Participants in the Project LifeSkills intervention had a 39.8% greater mean reduction in condomless sex compared to participants receiving standard care.

The FDA has granted a rare pediatric disease designation to CLR 131 for the treatment of Ewing’s sarcoma.

The device is used in conjunction with cataract surgery for patients with mild to moderate primary open-angle glaucoma.

If approved in early 2019, the nasal spray would become the first approved therapy indicated for seizure clusters in 17 years.

The FDA has granted an orphan drug designation to MeiraGTx Holdings Plc’s AAV-CNGA3 for the treatment of achromatopsia caused by mutations in the CNGA3 gene.

New research outlines the role of neutrophil cytoplasts in severe asthma which could lead to new therapeutic pathways to treat the disease.

These mistakes can be as simple as a worker touching their face before removing contaminated gloves.

A small number of cases of neural tube defects in babies born to mothers who were taking dolutegravir at the time of conception are prompting expanded study of the use of the drug in pregnant women.

Troy Trygstad spoke with MD Magazine® about the upcoming flu season and emphasized how different clinicians and parts of the healthcare system can play a role in promoting immunization.

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

Over time, it has become evident that suicide involves a complex mix of genetics, temperament, circumstances, external support, and access to means.

The FDA has approved migalastat (Galafold), the first oral medication for the treatment of adults with Fabry disease.

The Annovera combines segesterone acetate and ethinyl estradiol to prevent pregnancy and has a 2-4% failure rate.

Patisiran also makes history as the first FDA-approved small interfering ribonucleic acid treatment, a class of drugs that silences the portion of RNA involved in causing the disease.

Natural Cycles requires women enter a daily basal thermometer body temperature to detect minor rises in temperature at the time of ovulation. It also tracks the user’s menstrual cycle.

The FDA has approved patisiran infusion for the treatment of peripheral nerve disease caused by hereditary transthyretin-mediated amyloidosis in adult patients.

In a systematic review and meta-analysis, low-quality drugs are most prevalent low- to middle-income countries, and can cost up to $200 billion annually.

A new study has found that patients living with alkaptonuria, a highly rare genetic disease, can halt and partially reverse some symptoms with the drug nitisinone.

Investigators find boys treated with alkylating agents or with hydroxyurea for sickle cell disease resulted in a significant reduction in spermatogonial cell counts, suggesting that these agents can affect fertility.

A study evaluating a home and community education program to improve asthma management among Head Start students with asthma found that a majority of families have insufficient availability of asthma medications in the home.

The new test compares favorably with other well-established, reference molecular methods and can be performed as an on-demand test by all laboratory shifts with final test results available within 20 minutes.

According to pooled data, 91.8% of the hospitalizations due to firearm injuries in pediatric and adolescent patients were of urban patients, 4.8% were of micropolitan patients, and 3.3% were of rural patients.

From 1999 to 2014, prevalence of opioid use disorder among women giving birth increased 333% nationally, according to a report from the Centers for Disease Control and Prevention.

Diacerein 1% ointment (CCP-020) received a fast track designation from the FDA for the treatment of epidermolysis bullosa simplex.

A review of tardive dyskinesia epidemiology implicates newer "atypical" antipsychotics as well as older neuroleptics.

The novel methylphenidate formulation indicated for evening administration in patients with ADHD aged 6 years and older has shown improvement in the severity of ADHD symptoms in the early morning and throughout the day for patients.

This is the first approval for a drug preventing malaria in 18 years. Tafenoquine is taken beginning with an initial dose before entering a malaria-endemic area and subsequently once a week.

Krystal Biotech’s KB105 has received an orphan drug designation by the FDA for the treatment of patients with transglutaminase 1 deficient autosomal recessive congenital ichthyosis.

The FDA has granted an orphan drug designation to NSI-189 for the treatment of patients with Angelman syndrome.