
The US FDA has granted an orphan drug designation to Cellectar Biosciences, Inc.’s CLR 131 for the treatment of pediatric osteosarcoma.

The US FDA has granted an orphan drug designation to Cellectar Biosciences, Inc.’s CLR 131 for the treatment of pediatric osteosarcoma.

Investigators will assess the efficacy, safety, and tolerability of NanoFlu doses and formulations—both adjuvanted and unadjuvanted—in comparison to a pair of US marketed vaccines.

Of patients with a baseline blood eosinophil count of 300 cells per mL or more who were given benralizumab every 8 weeks, 75% were exacerbation-free during their second year of treatment.

A recent study investigating the HSPG2 gene in connection with tardive dyskinesia sheds new light on the root causes of the condition.

The findings lend to investigators believe that earlier endpoints for AMD diagnosis are needed.

When expectant mothers started antiretroviral therapy before pregnancy, their HIV-exposed but uninfected infants had lower infection risk than infants whose mothers started ART during pregnancy.

Electronic medical records alerts about patients at risk for C difficile infection seemed to increase the incidence, but investigators pointed toward improved detection as the culprit.

Pediatric traumatic brain injuries were found to be associated with a variety of health conditions and states with greater levels of private insurance and insurance adequacy.

The FDA has granted an orphan drug designation to Myelo Therapeutics GmbH’s Myelo001 for the treatment of acute radiation syndrome.

In animal models, not only did the drug help protect apelin function-deficient models from laser-induced choroidal neovascularization, but it did it through unique mechanisms that were independent of VEGF function.

The FDA has approved duvelisib for the treatment of adult patients with relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma after at least 2 prior therapies and adult patients with relapsed or refractory follicular lymphoma after at least 2 prior systemic therapies.

Investigators have found that the higher the symptom burden, the greater the detrimental impact on work productivity and daily activity for patients with myeloproliferative neoplasms.

The PrEPmate support intervention increased visit attendance and PrEP adherence among young men who have sex with men.

Female sex, previous gastrointestinal, and non-gastrointestinal comorbidities are associated with C difficile infection in women with UTIs, study finds.

The Eluvia stent system was approved based on data from the IMPERIAL non-inferiority trial, which compared Eluvia to Zilver PTX.

A comparative study found that online prevention programs are just as effective and have higher participation rates compared to in-person programs.

Tumor regressions were observed in 2 of the first 4 evaluable patients in the ongoing phase 2 trial evaluating DPX-Survivac in combination with low dose cyclophosphamide and pembrolizumab.

Pivotal clinical trial costs increased if more patients were needed to document treatment benefit, if active drug comparators were used, or to measure clinical end points rather than a change in a surrogate outcome.

The capsules of icosapent ethyl, which is derived from fish, showed a 25% relative risk reduction compared to placebo.

The Novartis humanized single-chain antibody fragment previously showed improved best corrected visual acuity in patients with nAMD versus aflibercept.

A new study by investigators in Denmark finds that children who develop acute lymphoblastic leukemia may be born with immune systems that are different from those of other children.

New research finds two-thirds of gay and bisexual men who know about pre-exposure prophylaxis have a positive view of it. The challenge is that only 4% are using it.

Investigators focused on adolescent African American patients, who notably report difficulties in handling environmental triggers and asthma management.

Lucia Novak discusses the importance of testing for cardiovascular outcomes of diabetes treatments and compares the cardiovascular effects of several diabetes drugs.

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

Lucia Novak, a diabetes care specialist, discusses some of the gaps that remain in diabetes management and what providers can do to help bridge those.

A software correction is currently being developed to address its inability to start therapy for patients at altitudes above 3200 feet.

Patients treated with the RECELL Systems were required to donate 97.5% less skin than standard-of-care, and reported statistically significant reduction in patient-reported pain, increases in satisfaction, and improved outcomes in donor scars.

Pauline Funchain, MD, highlights how humility may be one of the best characteristics in medicine right now.

Over 12 months of follow-up in the PROOF registry, pulmonary function remained largely stable in patients with idiopathic pulmonary fibrosis who received pirfenidone treatment.