Giuliana Grossi

Respiratory symptoms are significant among individuals with a history of smoking tobacco even without airflow obstruction.

Novel insights into the risk-benefit profile of nintedanib in children and adolescents with fibrosing interstitial lung disease (ILD) were provided by late-breaking data presented at ERS 2022 regarding the InPedILD clinical trial.

Late-breaking data presented at ERS Congress 2022 show that inhaler sensors and mobile health (mHealth) applications combined with pharmacist counseling improved treatment adherence for patients with uncontrolled asthma.

According to recent research, there’s no association between insomnia and vitamin D deficiency, although, insomnia did correlate with abnormal anxiety and depression scores among university students.

These data support web-based self-management for individuals with COPD with an objective, clinically relevant, short-term increase in physical activity levels compared to usual care.

CDC investigators stated that the expansion of telehealth strategies is “urgently needed” to provide medications for opioid use disorder because it could increase retention in care and improve outcomes for OUD.

“From this study it makes sense that the (icenticaftor) 300mg dose has a benefit-risk profile to further study the clinical benefits,” Dr. Frits Franssen, lead investigator, said at ERS Congress 2022.

Recent research from the National Psoriasis Foundation (NPF) found that 1 in 3 individuals living with psoriatic conditions experience depression.

In the second portion of the Rare Disease Report: CDKL5 podcast, Elia Pestana Knight, MD, a pediatric epileptologist shares her clinical expertise on CDKL5 deficiency disorder (CDD).

A novel biomarker was identified for posterior uveitis that postulates previously undetected sources of green and red emission fluorescent components (GEFC/REFC).

According to new research, stroke occurrence trends coinciding with tuberculous meningitis (TBM) are suggested to have regional characteristics.

Olipudase alfa (Xenpozyme) was developed by Sanofi to treat symptoms unrelated to the central nervous system in pediatric and adult patients with the life-threatening condition.

The FDA granted priority review for the Bioloigics License Application (BLA) Sanofi submitted for efanesoctocog alfa indicated for individuals with hemophilia A with a decision date set for February 28, 2023.

In the first episode of a 2-part Rare Disease Report, Whitney Mitchell, mother and caregiver to her 5 year old daughter Havilah, shared her experience leading up to, and now living with, Havilah’s CDKL5 deficiency disorder diagnosis.

Pemigatinib (Pemazyre) becomes the first and only targeted treatment for adults with extremely rare and aggressive blood cancer.

Severity of disease in patients with PAH coincided with cfDNA concentrations according to an NIH study with data from 2 independent cohorts.

Pfizer’s bivalent respiratory syncytial virus (RSV) prefusion F vaccine candidate demonstrated 85.7% efficacy in older patients with severe disease according to the phase 3 study.

New research supports the inclusion of sleep disturbance in clinical evaluation and management in the treatment of Phelan-McDermid Syndrome (PMS) due to the drastic impact it can have on the quality of life for patients and their families.

While investigators acknowledged the need for more high-quality, large-scale trials, cognitive behavioral therapy (CBT) serves as a promising intervention for insomnia in patients with cancer.

The investigational treatment SYNB1353 received FDA Fast Track Designation for homocystinuria (HCU), a rare metabolic disorder.

The investigational therapy is being evaluated in a phase 2 study for the reduction in risk of major thrombotic cardiovascular events in patients with ESRD on hemodialysis.

Investigators found that body surface area involvement on admission, malignant neoplasm, and connective tissue disease were independent factors associated with leukopenia in SJS/TEN.

Results from a new study highlighted the importance of achieving normal, stable glucose levels for individuals with type 2 diabetes.

Dermatologists addressed the overprescribing of antibiotics while acknowledging the necessity of antibiotics. Excessive prescribing practices contribute to the emergence of antibiotic resistance and furthers a patient's risk for resistant infections and treatment failures.

Data from this phase 2 study revealed that the efficacy and safety results support the continued investigation of mitapivat as treatment for adults with either α- or β-thalassemia who are not dependent on transfusions.

Among flares identified in outpatient (53%), inpatient (36%), and emergency department (9%) settings, the most utilized treatments were topical corticosteroids (35%), opioids (21%), and other oral treatments.

A high calorie, high fat diet is needed for patients with cystic fibrosis (CF) to maintain a normal weight resulting in an emphasized focus on their nutrition and weight management.

Investigators identified a correlation between severity of kidney-urinary tract complications and the affected protein involved with intermediate junctional epidermolysis bullosa (JEB).

The implementation of 13-valent pneumococcal conjugate vaccine (PCV13) was associated with an important reduction in the incidence of acute chest syndrome in children with sickle cell disease.

Despite their tendency to present with more severe psoriasis compared with other races and ethnicities, Asian patients receive significantly less time with a dermatologist.