News

In a trio of phase 3 studies, omadacycline met the FDA-designated primary and secondary efficacy outcomes while reporting consistent safety measures and tolerability.

Mutations in the MARK3 gene has been identified as the responsible culprit for pediatric blindness caused by a recessive genetic disorder.

The potassium chloride oral solution was approved through the new approval pathway for drugs with no other approved competitors.

The FDA has approved mogamulizumab-kpkc (Poteligeo) injection for intravenous use for the treatment of adult patients with relapsed or refractory mycosis fungoides or Sézary syndrome after at least 1 prior systemic therapy.

The FDA’s Antimicrobial Drugs Advisory Committee voted 12 to 2 in favor of the safety and efficacy of ALIS (amikacin liposome inhalation suspension) for difficult-to-treat patients with nontuberculous mycobacterial lung disease.

For patients with acute coronary syndrome and depression, taking escitalopram to treat depression resulted in significantly fewer major cardiac events compared to patients given placebo.

The REGENXBIO drug's promising phase 1 results has led to the addition of a fourth patient dosing cohort and a phase 2 trial initiation.

The FDA granted an orphan drug designation to Alector’s AL001, a human recombinant monoclonal antibody, for the treatment of patients with frontotemporal dementia.

A retrospective study showed a probability of maintaining HIV viral suppression of 98.2% at 48 weeks and 95.1% at 96 weeks, with the switch to the dolutegravir/lamivudine antiretroviral regimen.

Researchers were surprised to see the relatively high rates of secondhand smoke-related respiratory symptoms, such as shortness of breath and wheezing, among adolescents without asthma.

The FDA’s Antimicrobial Drugs Advisory Committee voted 12 to 2 in favor of the safety and effectiveness of ALIS for adults with NTM lung disease caused by MAC.

The FDA has approved lumacaftor/ivacaftor to include use in children ages 2 through 5 years with cystic fibrosis (CF) who have two copies of the F508del-CFTR mutation, making it the first medicine approved to treat the underlying cause of CF in this population.

The lack of higher reported mortality rates in states with a greater proportion of black residents suggests underdiagnosis of amyloidosis, including cardiac forms of the disease, in many areas of the United States.

The US Food and Drug Administration (FDA) has granted a rare pediatric disease designation to Prometic Life Sciences Inc.’s PBI-4050 for the treatment of Alström syndrome.

The FDA has granted an orphan drug designation to Pliant Therapeutics, Inc’s anti-fibrotic lead compound, PLN-74809, for the treatment of patients with idiopathic pulmonary fibrosis.

The US Food and Drug Administration (FDA) has granted an orphan drug designation to Alteogen Inc.’s ALT-P7 for the treatment of patients with gastric cancer.

The symptoms of tardive dyskinesia and drug-induced parkinsonism can be remarkably similar, but an accurate diagnosis is critical when treatment for one condition may worsen the other.

If done ethically, the genetic database's collaboration with GSK could provide researchers what they really need to improve neurological care: the right patients in the right studies.

People who ate at least 1 serving of oranges every day had more than a 60% reduced risk of developing late-stage macular degeneration in that time period.

Dr. Inga Hofman Zhang from the University of Wisconsin School of Medicine provides an update on treating MDS in children.

Dr. Naval Daver from MD Anderson Cancer Center talks about the relationship between MDS and AML and discusses new and novel treatments for both diseases.

Dr. Antonio Risitano, a hematologist from the University of Naples, discusses the most current thinking on PNH and its treatment.

Dr. Eric Padrone, a hematologist from H. Lee Moffit Cancer Center, discusses current thinking on MDS/MPN overlap disorders and myelofibrosis.

Dr. Timothy Olson, a pediatric hematologist at Children's Hospital of Philadelphia, discusses the most current thinking on the treatment of children with aplastic anemia.

The FDA has granted a fast track designation to Nohla Therapeutics’ dilanubicel (NLA101) for patients with high-risk hematologic malignancies receiving an allogeneic cord blood transplant.

Women and men are more likely to survive when treated by female doctors compared to male doctors, but the difference in heart attack survival is greater for women.

Investigators have discovered a rare cell type, referred to as Foxi1+pulmonary ionocyte, which is thought to be a key player in the biology of cystic fibrosis.

Investigators find that once-daily oral administration of BCX7353 at a dose of 125 mg or more resulted in a significantly lower rate of attacks of hereditary angioedema compared with placebo.