News

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 oral therapy was previously approved for use in children aged 6 years and older with CF and 2 copies of the F508del-CFTR mutation, the most common genetic form of the disease.

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.

Researchers were able to rule out myocardial infarction in 30% of patients immediately, and another 25% of patients at 1 hour. By 3 hours, the assay had successfully ruled out MI in 83.8% of patients, versus the 80.4% ruled out under the conventional assay.

Two studies examine whether users of daily oral PrEP would switch to a long-acting injection with interesting results.

Meeting with a peer support worker to complete a personal recovery workbook reduced readmissions over a year for patients with mental health challenges.

How researchers are using stem cells and a greater patient population to improve on the invasive methods used to treat the common women's disease.

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

Merck’s new non-nucleoside reverse transcriptase inhibitor (NNRTI), doravirine, achieved viral suppression in 73.1% of patients, according to new data released by the company.

The DR market is expected to accrue a sum of $2,490 million by 2022, growing at a compound annual growth rate of 14.4% from 2014-2022.

Financial hardship in a substantial proportion of adult survivors of childhood cancer was found by investigators from St Jude Children’s Research Hospital, suggesting screening for financial problems in patients.

The New England Journal of Medicine has published the 48-week phase 3 clinical trial results that supported the FDA approval of L-glutamine oral powder for the reduction of acute complications of sickle cell disease in adult and pediatric patients.

A qualitative narrative analysis of 300-plus submitted essays led to answers as to what factors contribute to inclusive environments at work or education institutions.

A phase 3 trial to investigate lenabasum as a potential treatment for dermatomyositis, a rare and often fatal multisystem inflammatory autoimmune disease affecting muscle and skin, have been announced by Corbus Pharmaceuticals Holdings, Inc.