CBT Intervention for Insomnia Can Help Oncology Patients
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.
FDA Grants Fast Track Designation to Synthetic Biotic for Rare Metabolic Disorder
The investigational treatment SYNB1353 received FDA Fast Track Designation for homocystinuria (HCU), a rare metabolic disorder.
Novel Anticoagulant Therapy Receives FDA Fast Track Designation for End Stage Renal Disease
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.
Investigation Identifies Factors Associated with Leukopenia in SJS/TEN
Investigators found that body surface area involvement on admission, malignant neoplasm, and connective tissue disease were independent factors associated with leukopenia in SJS/TEN.
Glycemic Variability, High Glucose Increase Mortality Risk in Patients with Type 2 Diabetes
Results from a new study highlighted the importance of achieving normal, stable glucose levels for individuals with type 2 diabetes.
Dermatologist Aim to Define Antibiotic Stewardship
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.
Phase 2 Data Support Continued Evaluation of Mitapivat for Alpha, Beta Thalassemia
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.
Investigators Highlight Unmet Needs of Patients with Generalized Pustular Psoriasis
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.
Increased Risk of Eating Disorders, ARFID Among Cystic Fibrosis Population
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.
Kidney-Urinary Tract Involvement with Intermediate Junctional Epidermolysis Bullosa
Investigators identified a correlation between severity of kidney-urinary tract complications and the affected protein involved with intermediate junctional epidermolysis bullosa (JEB).
New Evidence of S Pneumoniae in Acute Chest Syndrome
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.
Research Reveals how Psoriasis Treatment Differs Based on Patient Race, Ethnicity
Despite their tendency to present with more severe psoriasis compared with other races and ethnicities, Asian patients receive significantly less time with a dermatologist.
Combination Therapy Receives FDA Approval for Endometriosis
FDA approved combination therapy relugolix, estradiol, and norethindrone acetate (Myfembree) for the management of moderate to severe pain associated with endometriosis.
CDC will Include New ICD-10-CM Diagnosis Code for a Rare Disease in Fall
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.
Upadacitinib Provides Greater Efficacy Compared with Dupilumab for Atopic Dermatitis
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.
New Guideline on Vaccination Recommendations for Patients with Rheumatic and Musculoskeletal Disease
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.
Nonprofit Research on Gene Therapy for Rare Ocular Diseases
President and CSO of Odylia Therapeutics discusses the gene therapy pipeline, research and differences in operating as a nonprofit biotech company.
Phototesting Can Provide More Personalized Care for Photoaggravated Atopic Dermatitis
Investigators believe these results could help improve disease recognition and diagnosis while supporting development of personalized prevention and therapy.
Mary McGowan: Pulmonary Sarcoidosis Listening Session with the FDA
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.
Ustekinumab Becomes First FDA-Approved Biologic for Pediatric Psoriatic Arthritis
The therapy targets both IL-12 and IL-23 cytokines offering a new option for children 6 years and older living with active psoriatic arthritis.
Investigating the Relationship Between Vitamin D and Incident Fractures
Vitamin D supplements are often recommended for the improvement of bone health, but investigators say the data are unclear whether they prevent fractures.
Overdose Death Rates Before and After Take-Home Methadone Policy
After March 2020, overdose deaths not involving methadone continued to increase whereas methadone-involved overdose deaths remained stable.
Decreased Length of Stay After Total Joint Arthroplasty Predicted by Sleep Quality
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.
Methodology for an Adaptable HCV Immunoassay
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.
The Integration of Gene Therapy for Rare Disease
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.
FDA Approves Belimumab for Children 5-17 with Active Lupus Nephritis
Until now, treatment options for children with lupus nephritis were mostly limited to non-selective immunosuppressants and corticosteroids.
The Ethical Responsibilities of Precision Medicine According to ACP
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.
Study Supports Capsaicin as Potential Pain Treatment for Patients with SCD
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.
Young, Married Women have the Largest Unmet Contraceptive Needs
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.
Increasing Overdose Death Rates are Highest Among Black, Indigenous Individuals
According to a new report from the CDC, as fatal drug overdoses increase across the board, the Black and Indigenous communities represent a significantly greater death rates.
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