News

The bidirectional spread of the strain between farm animals and humans may represent an unappreciated route disseminating antimicrobial resistance genes between humans and animals.

Phase 2 clinical and immunological data presented at the 44th Annual EBMT Meeting this morning revealed that galinpepimut-S is safe and effective in high-risk multiple myeloma.

Traumatic brain injury increases children’s likelihood of developing ADHD up to 7 years later.

Adults with obesity treated with semaglutide 0.4 mg lost up to 13.8% of body weight after 52 weeks, significantly more weight than those treated with placebo who lost 2.3%.

At the ENDO Annual Meeting in Chicago, Progenics presented biochemical tumor marker data from its pivotal Phase 2 trial of iobenguane I 131 (Azedra) in patients with pheochromocytoma and paraganglioma

The study that linked parkinsonism asymmetry to the severity of psychopathology is the first of its kind to examine the relationship.

The industry has reached multibillion-dollar status, but more US patients seeking international care are driven by burden than opportunity.

The anti-inflammatory proved superior to as-need bronchodilator for control of early stage asthma symptoms in preschool children.

Results from a study published in the Orphanet Journal of Rare Diseases this week suggest that the hereditary neuropathy with liability to pressure palsies, which has been assumed to be rare, might not be uncommon in the Korean population.

Results were consistent with ELARIS UF-I and reduced heavy menstrual bleeding versus placebo.

A newly discovered hepatitis C virus (HCV) subtype harbors resistance-associated mutations to direct acting antivirals.

On Wednesday, the first patient with primary hyperoxaluria (PH) was randomized into OxThera AB’s Phase 3 trial of OC5 (Oxabact).

Patients being transferred out of the intensive care unit encounter medication errors at a rate of 47.5%, although most are not harmful.

According to data, patients with neovascular AMD can continue their prescribed use of AP/AC medication without negative effects on AMD outcomes.

Patients with hospital-acquired sepsis had higher rates of congestive HF, cerebrovascular disease, need for mechanical ventilation and vasopressors, increased LOS, more ICU days, and higher mortality than those with community-acquired sepsis.

Hartmann Wellhoefer, M.D., head of medical affairs at Shire, recently sat down with Rare Disease Report to discuss the company's recent work for the rare disease community, particularly lysosomal storage diseases.

This morning, it was announced that the U.S. FDA has approved CSL Behring’s maintenance therapy for the treatment of patients with chronic inflammatory demyelinating polyneuropathy (CIDP).

Lead researcher Ben Seymour, PhD, noted that this brings medicine a step closer to harnessing the brain's natural pain relief system in a more specific way.

This is one of the first interventions to successfully reduce sexual risk among juvenile offenders on probation.

Adherence to generic multi-tablet regimens was high and comparable with adherence to brand multi-tablet regimens and to single tablets.

The market price for gene therapy greatly exceed the costs of the gene editing development and the equipment required for the task.

ALXN1210 has demonstrated non-inferiority to eculizumab in complement inhibitor treatment-naïve patients with paroxysmal nocturnal hemoglobinuria, according to results from a pivotal Phase 3 study.

The mortality rate in C. difficile-infected patients with acute kidney infection is decreasing, from 23.9% to 14.8%, but hospitalizations increased significantly by 1.8 over the study period.

The U.S. Food and Drug Administration has granted Fast Track designation to gaboxadol (OV101), a therapy in development for the treatment of Fragile X syndrome.

Offering PrEP at public health-care clinics in a middle-income setting can retain high numbers of participants and achieve high levels of adherence without risk compensation in investigated populations.

Abeona announced that the U.S. FDA has granted rare pediatric disease designation to ABO-202, a gene therapy in development for the treatment of infantile and late infantile-onset Batten disease.

The approval was granted based on efficacy and safety data from the Escort-HU study, which included 495 pediatric patients with sickle cell anemia.

The U.S. FDA has approved hydroxycarbamide/hydroxyurea tablets in pediatric patients suffering from sickle cell anemia, ages two years and older, France-based Addmedica has announced.

Elevated thyroid stimulating hormone was linked to female sex, higher BMI, liver fibrosis, AST, and low albumin.

New interim study results show women are open to using a vaginal ring for HIV prevention, but scientists are still trying to figure out when it works, and why.