Don Sapatkin

In-depth interviews and an online survey found support for gene therapy as a treatment for inherited blood disorders, but also several obstacles that must be addressed.

Up to half of patients showed significant improvement in study results presented at the American Academy of Ophthalmology (AAO) annual meeting

One of two new studies found most patients doing well six years after treatment. Another study found blood cancers in 17% of patients given the therapy using a slightly different protocol.

The Phase I/II trials for rare forms of hereditary blindness further confirmed the potential for gene therapy treatments of congenital diseases of the retina.

“Cure” is not an immutable term, the authors of a new study write, and its meaning depends on the context in which it is used. Sometimes, it shouldn’t be.

The researchers took what they said was a conservative approach when plugging variables such as one-time and future costs into their updated model.

As supplemental screening for breast cancer becomes more common, the modeling study supplies long-term projections of how many deaths could be averted but also how many false-positive biopsy recommendations would be added.

CMS will provide key logistical support to states that participate in its outcomes-based funding model for gene therapy treatment for sickle cell disease.

Gene therapy is enormously complicated, inefficient and slow. Early tests of a newly developed machine learning model show how much faster, and more efficient, it might be.

The key to lowering gene therapy costs – and getting treatments to where they are most needed – could be making them in middle- low-income countries, argue the authors of a recent commentary in Nature.

The authors say their findings should not change women’s calculations about double, or bilateral mastectomy, which physicians generally discourage.

Respiratory syncytial virus (RSV) is the leading cause of bronchiolitis, which accounted for nearly one-fifth of all pediatric hospitalizations in the U.S. before the pandemic.

The intent is for both parties to share information that will enhance the agency’s efficiency and quality of reviews.

Denmark has also set up a reimbursement arrangement for Hemgenix, which has a list price of $3.5 million in the U.S.

Gene therapies are incredibly expensive and tend to benefit small numbers of people, a combination that presents myriad financing challenges for patients, providers, payers and producers. A new study explored some of the options.

Monday’s action followed by one month the agency’s approval of another first — a monoclonal antibody immunization for newborns — against respiratory syncytial virus, the leading cause of infant hospitalizations in the United States

A new study has found that magnesium sulfate given between 30 weeks and 34 weeks’ gestation did not lower the incidence of cerebral palsy in premature infants.

The Centers for Disease Control and Prevention cleared the way for the first immunization approved to protect all young infants and at-risk older babies against respiratory syncytial virus (RSV). There are questions, though, about when insurance coverage will kick in.

A huge study in Sweden found that the risk was greatest when both parents had been diagnosed with any of a large range of mental illnesses.

A large prospective study found large, statistically significant effects of heartburn on sleep quality.

A demonstration project showed that frequent testing reduced the median time to diagnosis by nearly 14 months.

Leaving the room because of how a partner sleeps is surprisingly common, a survey found. People make all sorts of other accommodation, too.

Between 9% and 12% of American adults have insomnia, and 85% of them also are diagnosed with comorbid conditions like type 2 diabetes and dementia. While the numerous health consequences of comorbid insomnia have been well-documented, the additional costs for specific disease groups have not been measured in large studies — until now.

Most babies born to mothers who used opioids during pregnancy are delivered with a form of addiction called neonatal abstinence syndrome. It is treated in the hospital, but how long the treatment lasts depends on the severity of withdrawal symptoms called neonatal opioid withdrawal syndrome — the amount of distress that the newborn is experiencing.

Standard guidelines that insomnia treatment for people with alcohol use disorder be delayed until abstinence is achieved delays therapy for many people — and most patients still struggle with sleep even after successfully quitting alcohol.

Some effects of poor sleep (fatigue, daytime dysfunction) resemble some symptoms of long COVID. As it turns out, infected women who were healthy sleepers before and early in the pandemic were less likely to report long COVID symptoms.

Respiratory syncytial virus (RSV) can be deadly for the very young and the old and frail. GSK's vaccine will be marketed under the brand name Arexvy and Pfizer's under the name Abrysvo.

The purpose of the federal vaccine safety reporting system often is misunderstood, and its data can be easily used to spread false information about vaccination. A new report from the Annenberg Public Policy Center at the University of Pennsylvania looks at how it was manipulated to raise doubts about COVID-19 vaccines and suggests remedies.

In one of the largest and longest-lasting studies of workplace culture and employees’ sleep patterns, Scandinavian researchers found both a clear link between the two and improved sleep over time when leadership increased psychosocial resources.

Seasonal influenza kills hundreds of thousands of people every year due to a constantly changing virus and highly inefficient vaccine production. A universal flu vaccine could deal with the first problem, and mRNA technology with the second.