News

The FDA has granted a rare pediatric disease designation to Krystal Biotech’s KB105 for the treatment of patients with TGM-1-deficient autosomal recessive congenital ichthyosis.

Use of this new approach suggests that HCV infection burden in New York City has been underestimated in populations considered high-risk for HCV, and that HCV transmission has increased among young adults in the city.

Phase 3 data for ibalizumab showed that nearly half of patients had a reduction in viral load of more than 80% after 25 weeks of therapy.

In a clinical trial, the expedited Transcranial Magnetic Stimulation device produced a patient response/remission rates of 32% depressive symptom improvement rates of 49%.

Oxervate eye drops, which contain cenegermin, are the first approved treatment for a rare condition that affects fewer than 5 in 10,000 individuals.

The FDA approves the first drug, cenegermin, for the treatment of neurotrophic keratitis, a rare disease affecting the cornea.

Investigators have discovered a mechanism that could reduce the toxic aggregation of huntingtin protein, which is responsible for neurodegeneration in patients with Huntington’s disease.

A potential first-in-class, oral medication to treat uterine fibroids has been accepted by the FDA.

The new test should replace the water deprivation test as the new standard of care, investigators say.

In an analysis of 118 clinical trials since 2001, investigators found females and minorities comprised just 27% and 22% of the total patient population, respectively.

Results from a 12-month extension study show that elagolix taken with hormone therapy reduced menstrual blood loss in 87.9% of women with uterine fibroids.

The study authors state these data can be used to develop a tailored approach to the elimination of hepatitis C virus infection, worldwide.

The results of a new meta-analysis revealed metronidazole was among the worst choices for treating the infection.

A team of investigators at Cleveland Clinic discover a novel subtype of multiple sclerosis which features neuronal loss but no demyelination of the brain’s white matter.

Cleveland Clinic investigators have discovered a novel form of the neurological condition which does not demyelinate the brain's white matter.

The FDA grants RMAT designation to AT132 for the treatment of patients with X-linked Myotubular Myopathy.

Stiripentol (Diacomit) has been approved for treatment in patients 2 years of age and older with Dravet syndrome, a rare form of epilepsy.

A rapid genetic diagnostic tool coupled with a sustained release therapeutic is being developed for lower-grade gliomas by a team of investigators.

The Luminelle DTx Hysteroscopy System can be used in-office by physicians to visually assess suspicious tissue in the uterus and allow for a visually-aided biopsy.

The FDA has approved stiripentol (Diacomit) for the treatment of seizures associated with Dravet syndrome, a rare form of epilepsy.

Compared to those with LDL cholesterol levels 160 mg/dL had a 70-90% increased risk.

The dual use of ribavirin and direct-acting antivirals during treatment for hepatitis C virus was found to independently predict decline in mental health-related quality-of-life.

The results of a new study indicate that most physicians are not following the CDC’s recommendation to screen all Baby Boomers for hepatitis C.

The FDA has approved pembrolizumab to be used in combination with pemetrexed and platinum as first-line treatment for patients with metastatic, non-squamous non-small cell lung cancer, with no EGFR or ALK genomic tumor aberrations.

The FDA has granted an orphan drug designation to Immune Pharmaceuticals, Inc.’s bertilimumab for the treatment of bullous pemphigoid.

The FDA accepts Alexion Pharmaceutical Inc.’s BLA for review for approval of ALXN1210 for the treatment of patients with paroxysmal nocturnal hemoglobinuria.

The FDA has granted an orphan drug designation to ASLAN Pharmaceuticals’ ASLAN003 for the treatment of acute myeloid leukemia.

The FDA has granted a fast track designation to gene therapy product candidate AAV-CNGB3 for the treatment of achromatopsia caused by mutations in the CNGB3 gene.

The FDA has approved Bristol-Meyers Squibb Company’s nivolumab (Opdivo) for the treatment of metastatic small cell lung cancer (SCLC) that has progressed after platinum-based chemotherapy and at least 1 other line of therapy.

Quebec-based investigators have highlighted the central role of bridge symptoms in the developmental process leading to long-term anxiety.