News

A phase 3 trial demonstrates that tafamidis significantly reduced the combination of all-cause mortality and cardiovascular-related hospitalizations in patients with ATTR-CM.

The FDA has granted an orphan drug designation to ReflectionBio's AAV-based gene therapy, the RBIO-101 program (AAV.CYP4V2), for the treatment of Bietti's crystalline dystrophy (BCD).

In 2016, US firearm suicides represented 35.3% of all global firearm suicides—despite the US just comprising 4.3% of the global population.

Early trials have shown promising results for hemophilia therapies and now could be the most promising time for a lasting treatment and possible cure.

Results of an analysis indicate suicidal behavior occurred more often in patients taking an efavirenz-based treatment for HIV than in a control group that had not started treatment.

What a longtime family practitioner in a state that authorizes medical aid in dying has learned through his own experience of the controversial practice.

New insights might help researchers improve the efficacy of LTA4H inhibitors.

Relevant cIAI diagnoses include intra-abdominal abscess, stomach perforation, peritonitis, appendicitis, and more. Eravacycline's efficacy and safety were established in non-inferiority studies comparing it to the widely-used therapies ertapenem and meropenem.

The FDA has granted a fast track designation to CX-01 for the treatment of patients over 60 years of age receiving induction therapy for newly-diagnosed AML.

The FDA has granted a rare pediatric disease designation to AAV-CNGA3 for the treatment of patients with achromatopsia due to mutations in the CNGA3 gene.

As more physicians are caring for transgender patients, many are finding they are unprepared and they lack the training, skills, and resources to do so, according to the authors of a recent review article.

The FDA has approved ibrutinib (IMBRUVICA) plus rituximab (RITUXAN) for the treatment of adult patients with Waldenström's macroglobulinemia, a rare and incurable type of non-Hodgkin's lymphoma.

Patients whose direct-acting antiviral treatment failed are finding hope in a new combination of 3 existing hepatitis C virus drugs.

A new survey found that Clostridium difficile infections have a substantial impact on the health-related quality of work, work life, and physical activity of those who are infected.

In a 15,000-plus patient analysis that spanned more than 7 years, aspirin reduced vascular risks in patients—but also increased their rate of major bleeding events.

Patients administered the FDA-approved therapy reported tripled rates of weight-loss, along with noninferiority for risk of major cardiovascular events, over 1 year and an extension period.

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

The update reported significant improvements in future cardiovascular risks in patients at risk for chronic angina initially referred for CTA plus standard care, versus those only referred to standard care.

Altreno is a lotion containing .05% tretinoin, a retinoid that reduces inflammation and lesions associated with acne vulgaris.

A contributing mechanism responsible for the development of idiopathic pulmonary fibrosis has been identified.

The FDA grants an orphan drug designation to a novel gene therapy candidate for the treatment of patients with hereditary angioedema.

The phase 2b dose-ranging trial will involve 216 patients across 12-15 research sites in North America and Europe, beginning in the UK later this month.

The first darunavir-based single tablet regimen for HIV has shown positive results in trials as both a switch therapy and for the rapid initiation of treatment in those who are newly diagnosed.

Ridinilazole caused less microbiota disruption, and investigators believe it may be superior to vancomycin for preventing Clostridium difficile infection recurrence.

Lanadelumab (Takhzyro) has been approved for the treatment of hereditary angioedema in patients 12 and older.

The FSA approves lanadelumab (Takhzyro), the first monoclonal antibody approved in the United States to treat patients 12 years and older with types I and II hereditary angioedema.

The Kala Pharmaceuticals therapy INVELTYS becomes the first twice-daily ocular corticosteroid indicated for such treatment.

Howard Levy, MBBCh, PhD, MMM, highlights phase 2/3 trial of MarzAA for the treatment of hemophilia.

A type of electrical stimulation directed at the brain and spine may help treat the symptoms of rare movement disorders like neurodegenerative ataxias.

Research indicates that rates of Clostridium difficile infection among those typically not considered at high risk of infection are increasing, and these infections are becoming more frequent, severe, and difficult to treat.