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Gene Therapies for Hemophilia. Possibly Curative, Likely Ultraexpensive

Hemophilia is a rare, X-linked, hereditary bleeding disorder characterized by blood clotting factor deficiencies that affects mainly males. It results in excessive bleeding, particularly in the joints, soft tissues and muscles. The bleeding usually happens after internal or external trauma, but in rare cases, it occurs spontaneously. According to the CDC, up to 33,000 males in the United States live with hemophilia, but the condition can be fatal. Hemophilia is divided into subtypes, the two mos

Anxiety, Depression, and Diabetes Higher in Older Males with Hemophilia; Cardiovascular Disease Lower

Anxiety, Depression, and Diabetes Higher in Older Males with Hemophilia; Cardiovascular Disease Lower Lack of blood clotting may afford some protection against common cardiovascular disease events, such as ischemic stroke. People living with hemophilia now can expect a longer lifespan than those who had the disease decades ago. Thanks to new advances in treatment, people with hemophilia now have an average life expectancy close to that of people without hemophilia. However, living into older a

For Plaque Psoriasis Treatment, Abundance Gets More Company

Note: This story was updated on Aug. 19 People with plaque psoriasis have a number of treatment options: topical agents, phototherapy, oral treatments and biologics. And now drugmakers are vying to create even more. In front of the queue is Vtama (tapinarof) cream 1%, the first nonsteroidal topical agent approved to treat plaque psoriasis. Developed by Dermavant Sciences, a biotech company in Morristown, North Carolina, Vtama is a cream containing an aryl hydrocarbon receptor agonist. Aryl hyd

Why Steroids Don’t Work in Severe Asthma Patients

Over 25 million people in the United States live with asthma, and up to 10 percent of these hve severe asthma, according to the American Lung Association. In most people with asthma, inhaled corticosteroids are effective as rescue and maintenance therapy in decreasing airway swelling and irritation. However, people with severe asthma fail to respond to inhaled corticosteroids. Until recently, the reasons behind this poor response had been a perplexing mystery. In a study published earlier this

Cost Effectiveness Analysis of Biomarkers for Guiding Asthma Treatment in Children

Current Global Initiative for Asthma (GINA) guideline for asthma management recommends periodic evaluation of airway inflammation to prevent hospitalization in people with asthma. Additionally, monitoring airway inflammation provides a valuable tool for predicting asthma exacerbations, which in turn aids in optimizing the use of biologics and corticosteroid treatments. Although various methods exist to conduct this assessment, noninvasive procedures tend to be more feasible in pediatric patient

American Academy of Family Physicians Awarded $31 Million for Comparative Asthma Treatment Study

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. Penn State College of Medicine will partner with the AAFP, serving as the data coordinating center for the study. The Individualizing Treatment for Asthma in Primary Care (iTREAT-PC) study will enroll 3,200 adults and adolescents 12 to under 76 years of age who have asthma. The study will be con

Albuterol-Budesonide Combination Moves Through the Asthma Pipeline

The FDA has accepted a New Drug Application (NDA) filed by AstraZeneca and British drug maker Avillion for their potential first-in-class combination short-acting beta2-agonist (SABA) and corticosteroid inhaler. PT027 combines albuterol and budesonide in a fixed-dose inhaler with a proposed indication of as-needed treatment or prevention of bronchoconstriction and prevention of exacerbations in adults and children ages 4 years and older with asthma. The NDA submission was based on results from

Dupixent Combined with House Dust Mite SLIT May Help Improve Control in Allergic Asthma

Allergen-specific immunotherapy (ASI) has been used as a disease-modifying treatment to relieve symptoms in patients with IgE-mediated allergic conditions, including allergic asthma. House dust mites (HDMs) are a significant trigger in allergic asthma, and HDM sublingual immunotherapy (SLIT) has been effective in improving symptoms and reducing exacerbations in patients with asthma. The Global Initiative for Asthma (GINA) and European Academy of Allergy and Clinical Immunology (EAACI) guideline

Fluctuations in Sex Hormones May Drive Asthma Symptoms in Women

Fluctuations in Sex Hormones May Drive Asthma Symptoms in Women 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. In childhood, asthma is more prevalent in boys than in girls (11.9% vs. 7.5%). However, at puberty, the trend flips. In adults, the incidence of asthma in men is 6.3% versus 9.6% in women. Additionally, women with asthma are more likely to have severe a

A Surge of Biologics for Severe Asthma

Six biologics on the market and more are in the pipeline. Biologics target the inflammatory cytokines involved in the pathogenesis of asthma. Asthma is a chronic respiratory disease that causes narrowing and inflammation of the airways that compromises breathing and, in some cases, can be fatal. Symptoms include coughing, wheezing, shortness of breath, chest tightness and chest pain. Nearly 25 million people in the United States have asthma, and 10 people die from the disease daily. Current ast

IL-13 in People with Allergic Asthma May Offer Some Protection Against Severe COVID

Many people with chronic respiratory conditions are at high risk of developing severe disease from infection with SARS-CoV-2, the virus that causes COVID-19. However, epidemiologists have found that individuals with allergic asthma are a peculiar exception. They are less susceptible to severe COVID than the general population. Allergic asthma is a type of severe asthma typically triggered by allergens, such as pet dander, mold, and pollen. Individuals with this type of asthma have high levels o

Follow-up Care Helps Prevent Repeat Asthma-Related Emergency Department Visits in Children and Young Adults

According to the Centers for Disease Control and Prevention (CDC), asthma is the most common chronic lung disease in children. It affects about 6 million children in the United States and accounts for over 500,000 emergency department visits for children each year. Emergency care is associated with increased costs for families and health care systems as well as significant disruptions to children’s learning via missed school days. Clinical practice guidelines for asthma management recommend tha

Medicaid Covers Nonmedical Services as Part of Initiative to Address Social Determinants of Health

Medicaid Covers Nonmedical Services as Part of Initiative to Address Social Determinants of Health California’s “in lieu of services” (ILOS) program includes asthma remediation services such as dehumidifiers, improved ventilation and mold removal. Children with severe asthma living in low-income homes are disproportionately affected by environmental factors that can trigger asthma exacerbations. Frequently these children experience asthma complications requiring costly hospitalizations or emer

New Target Discovered for Non-Type 2 Inflammation in Severe Asthma

Severe asthma is categorized based on patients’ response to treatment and the presence of biomarkers, such as immunoglobulin E (IgE), eosinophils, and neutrophils. Allergic asthma, eosinophilic asthma, and non-eosinophilic asthma are types of severe asthma. Patients with allergic asthma typically have high levels of IgE when exposed to allergens. People with eosinophilic asthma have increased eosinophil levels, and those with noneosinophilic asthma show neither increased IgE nor eosinophil level

Dupixent Improves Severe Asthma Symptoms in a Real-World Setting, but Switching May Require Eosinophil Monitoring

Dupixent Improves Severe Asthma Symptoms in a Real-World Setting, but Switching May Require Eosinophil Monitoring Real-world study conducted in Japan shows Dupixent (dupilumab) reduced the number of annual severe asthma exacerbations b 53%, there were also adverse events associated with high eosinophil levels in patients who had switched from a different biologic before starting Dupixent. In 2018, Sanofi and Regeneron Pharmaceuticals were granted FDA approval for Dupixent (dupilumab) as add-on

Several Biologics To Treat Severe Asthma Can Be Self-Administered at Home — Patients Prefer It

Patients with severe asthma are often burdened with frequent exacerbations requiring numerous visits to clinics and doctors’ offices for symptom monitoring and treatment evaluation. New severe asthma treatments, specifically biologics, have helped manage severe asthma symptoms and decrease the frequency of exacerbations. In this respect, they have helped improve patients’ quality of life. However, many patients using biologics for severe asthma must receive treatment from a healthcare profession
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