Giuliana Grossi

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.

FDA approved combination therapy relugolix, estradiol, and norethindrone acetate (Myfembree) for the management of moderate to severe pain associated with endometriosis.

In October, a new ICD-10-CM diagnosis code will be activated for APDS, a rare primary immunodeficiency disease, which will enable physicians and payers to add the diagnosis to patient charts for the first time in the US.

Both treatments showed tolerable safety as in the phase 2 and 3 trials, but when evaluated in four body regions on patients, upadacitinib surpassed dupilumab in all of them.

The new treatment guideline addressed the possibility of differing safety and effectiveness outcomes among patients with rheumatic and musculoskeletal disease regarding vaccination compared with the general population.

President and CSO of Odylia Therapeutics discusses the gene therapy pipeline, research and differences in operating as a nonprofit biotech company.

Investigators believe these results could help improve disease recognition and diagnosis while supporting development of personalized prevention and therapy.

Prior to the launch of FSR's webinar on pulmonary sarcoidosis, Mary McGowan, CEO, discusses the private listening session held with the FDA that addressed the critical needs in the field.

Vitamin D supplements are often recommended for the improvement of bone health, but investigators say the data are unclear whether they prevent fractures.

After March 2020, overdose deaths not involving methadone continued to increase whereas methadone-involved overdose deaths remained stable.

Patients with high preoperative sleep quality, high preoperative albumin level, free of digestive disease and undergoing total hip arthroplasty procedure are more likely to be discharged within 2 days as opposed to 3.

Investigators articulated the development and validation of an enzyme-linked immunoassay against hepatitis C virus (HCV). They suggest the implementation of this low cost assay among at-risk populations.

There’s still much to be understood about gene therapy and its application to rare diseases like sickle cell, but at the progressive pace it’s moving at, gene therapy is likely to be integrated further into standard practice.

The American College of Physicians (ACP) aimed to inform on the ethical decision making regarding the integration of precision medicine and genetic testing into clinical care.

Investigators found that when assessing patients with sickle cell disease and neuropathic pain the TRPV1 receptor is a crucial component in the development of hypersensitivity and potential therapeutic benefit of capsaicin.

According to the most comprehensive assessment of worldwide contraceptive use, vast differences in the types of contraceptives that are utilized across regions and age groups were identified.