Rosanna Sutherby, Pharm.D.

Dozens of gene mutations associated with amyotrophic lateral sclerosis have been identified. Drug developers have homed in on a trio of genes.

Because vitiligo affects appearance, people with vitiligo are disproportionately affected by psychiatric conditions, including depression.

The melanocytes attacked by the autoimmune disease are present in the eyes and ears, not just the skin, so vitiligo can adversely affect vision and hearing.

German researchers developed a modified adeno-associated virus capsid that has greater affinity for liver cells.

Enyzyre says its at-home testing system will be a major advance for people with hemophilia because they will better able to manage the disease on their own.

CSL Behring's gene therapy, called EntranaDez, may be approved later this month. BioMarin, Pfizer, Bayer and Freeline Therapeutics also have gene therapies for hemophilia in development.

The tool successfully identified children with high asthma risk as young as 3 years of age. The screening tool was found to be more accurate at predicting asthma, persistent wheezing, and related emergency room visits and hospitalizations than standard screening and diagnostic tools.

Findings from research presented at the American College of Chest Physicians suggest the importance of selecting the right biologic from the start.

University of Wisconsin researchers find that people with asthma benefit from intervention that includes meditation and yoga and sustained focus on the breathing, thoughts, feelings, and bodily sensations.

Gilead’s magrolimab is the leading CD47 immune checkpoint inhibitor in development.

Several drugs with novel mechanisms of action have been put on the pathway for speedier approval decisions by the FDA. Phase 2 and 3 trials are underway.

Early interventions might preserve lung function, particularly in younger patients with frequent asthma exacerbations, say authors.

The hemophilia organization developed a shared decision-making tool for patients and providers.

Long-acting recombinant replacement factor gets favorable rating from multidisciplinary group. Gene therapy was viewed with some wariness.

Program in Hidalgo, Texas, cost less than $250 per family. A study found that the intervention saved each household an average of $36 per day.

A longitudinal study aims to identify which children with asthma are more likely to suffer a loss of lung function.

Findings suggest that screening people with hemophilia for diabetes and hypertension might have benefits.

An analysis found that a single dose of gene therapy would be more cost-effective than on-demand or prophylaxis factor replacement in 92% of cases.

Repurposing Gleevec as an asthma drug is one of the possibilities that the PrecISE trial of severe asthma is examining. The trial has an adaptive design.

Infants exposed to acid reducers may have an increased risk of developing asthma and recurrent wheeze in childhood.

Three gene therapies in late-stage development are designed to supply working copies of genes that people with hemophilia are missing. They may be one-time, curative treatments, but the seven-figure price tags will be an issue.

Lack of blood clotting may afford some protection against common cardiovascular disease events, such as ischemic stroke.

Nonsteroidal topical agents and TYK2 inhibitors may join an already full armamentarium.

Secretions of fibroblast growth factor (FGF) and granulocytic colony-stimulating growth factor (G-CSF) in airway epithelium may be to blame.

Using biomarkers for tailoring asthma treatment can reduce the frequency of asthma exacerbations. Some recent results suggest that eosinophil-guided asthma management is associated with lower annual costs than FeNO-guided management.

The Patient-Centered Outcomes Research Institute (PCORI) has approved an award for the American Academy of Family Physicians (AAFP) of $31 million to fund a large comparative study of asthma treatments.

MANDALA trial results showfixed dose combination of albuterol and budesonide was more effective than albuterol alone as rescue medication.

House dust mites after a common trigger for allergic asthma. Guidelines recommend house dust mite sublingual immunotherapy as add-on therapy for patients with house dust mite-related allergic asthma.

Asthma becomes more prevalent in females than males in puberty. Researchers have hypothesized that sex hormones may play a significant role in the manifestation of asthma symptoms.

Bispecific antibody therapies are also in development.