FAMILY HEALTH

Epic Bio, a biotechnology firm specializing in gene expression modulation through non-cutting dCas proteins, has secured Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for EPI-321 in treating facioscapulohumeral muscular dystrophy (FSHD), the most prevalent adult muscular dystrophy. EPI-321, designed to suppress abnormal DUX4 gene expression, will undergo a Phase 1/2 clinical study starting H1 2024 to assess safety and efficacy in FSHD patients. The FDA's designation provides financial benefits, including tax credits, fee waivers, and potential market exclusivity for 7 years upon approval. FSHD, affecting 4.6 in 100,000 globally, lacks a cure, and EPI-321 aims to address its molecular mechanisms through epigenetic therapy.

FDA Grants Orphan Drug Designation to Epic Bio's EPI-321 for Treating FSHD: Phase 1/2 Clinical Study to Begin 2024

GASTROENTEROLOGY

7-mm TIPS reduced hepatic encephalopathy while maintaining similar shunt function and rebleeding outcomes in cirrhosis.

7-mm TIPS Reduces Hepatic Encephalopathy in Cirrhosis

A multicenter randomized trial found that 7-mm covered TIPS reduced overt hepatic encephalopathy compared with 8-mm stents in cirrhotic patients with relatively small livers. Over 2 years, HE occurred in 21.4% vs 37.2%, while shunt dysfunction, rebleeding, and transplant-free survival were similar, supporting 7-mm TIPS as a potential option for variceal rebleeding prevention.

CARDIOLOGY

The Trilogy valve showed high procedural success and favorable outcomes in patients with symptomatic aortic regurgitation.

Trilogy Valve Shows Promise for Transcatheter Aortic Regurgitation

A multicenter European study of 363 patients with symptomatic aortic regurgitation found that transfemoral TAVR using the JenaValve Trilogy achieved 98% technical success and favorable valve performance. At discharge, 86% had none or trace leakage, while 88% were in NYHA class I–II at 30 days, supporting its potential for patients unsuitable for surgery.

PULMONOLOGY

Dexamethasone suppresses IL-1β and IL-13 signaling while promoting anti-inflammatory gene activity in airway smooth muscle.

Glucocorticoids Modulate Inflammation in Airway Smooth Muscle

RNA-seq and chromatin analyses of human airway smooth muscle cells reveal that dexamethasone suppresses gene activation driven by IL-1β and IL-13, key pathways in Type I and Type II airway inflammation. The findings suggest glucocorticoids primarily repress inflammatory signaling through transcriptional competition while also cooperating with NF-κB to activate anti-inflammatory genes.

Anti-TNF therapy may cause false-negative LRG1 results, limiting its accuracy for monitoring ulcerative colitis activity.

Anti-TNF Therapy May Limit LRG1 Accuracy in Ulcerative Colitis

A study found that anti-TNF therapy markedly reduced the ability of serum LRG1 to detect active ulcerative colitis. LRG1 accuracy fell from an AUC of 0.91 without anti-TNF therapy to 0.44 with treatment, with 45% of active cases yielding false-negative results. Anti-TNF use increased false-negative odds 9.3-fold, highlighting the need to consider treatment context when interpreting LRG1.

On-body risankizumab was highly accepted by Crohn’s disease patients and preferred over other treatment delivery methods.

On-Body Risankizumab Injector Highly Accepted in Crohn’s Disease

A prospective multicenter study found that the risankizumab on-body injector (OBI) was highly acceptable among 58 patients with Crohn’s disease, with acceptance remaining stable through 24 weeks. It was preferred over infusions, pen injectors, and syringes by 64% of patients. Mild application-site pain and erythema were the most common adverse events, with no treatment discontinuations.

New equations for PNIF and PNEF may improve objective nasal airflow assessment, pending external validation.

New Reference Equations Established for PNIF and PNEF

A cross-sectional study of 239 healthy adults developed reference equations for peak nasal inspiratory flow (PNIF) and expiratory flow (PNEF) using height, weight, and sex. PNIF showed greater homogeneity and lower variability than PNEF, supporting its potential as a complementary objective measure of nasal airflow. External validation is needed before wider clinical use.

DERMATOLOGY

PRP combined with fractional CO₂ laser may improve acne scars and satisfaction without increasing postoperative adverse effects.

PRP Plus Fractional CO₂ Laser Improves Atrophic Acne Scars

A meta-analysis of seven randomized trials involving 165 patients found that adding platelet-rich plasma (PRP) to fractional CO₂ laser treatment improved outcomes for atrophic acne scars. Combination therapy increased the likelihood of excellent improvement and high patient satisfaction without significantly increasing erythema, edema, or hyperpigmentation, although larger studies with longer follow-up are needed.

Higher dietary fiber intake was linked to lower COPD prevalence, inflammation, and circulating LPS in observational analyses.

Dietary Fiber Intake Linked to Lower COPD Risk and Inflammation

Analysis of NHANES data and a clinical cohort suggests that higher dietary fiber intake is associated with lower COPD prevalence and reduced systemic inflammation. In COPD patients, greater fiber intake was linked to lower inflammatory markers and circulating LPS, which were associated with poorer lung function. The observational findings warrant confirmation in prospective and intervention studies.

PEDIATRICS

Prenatal TGA diagnosis enabled timely intervention, with 88.8% neonatal survival and coronary anomalies linked to mortality.

Prenatal TGA Diagnosis Linked to High Neonatal Survival

A retrospective study of 116 fetuses with prenatally diagnosed d-transposition of the great arteries (d-TGA) found an overall neonatal survival rate of 88.8%. Coronary artery anomalies were strongly associated with mortality, occurring in 30.8% of non-survivors versus 4.9% of survivors. Prenatal diagnosis enabled planned tertiary-center delivery and timely prostaglandin and surgical management.

Biologic benefits in COPD varied by eosinophil levels, with greater efficacy generally seen in patients with elevated BEC.

Biologics in COPD: Eosinophil Levels May Predict Treatment Benefit

A systematic review of 16 publications covering seven biologics found that treatment benefits in moderate to very severe COPD varied by baseline blood eosinophil count (BEC). Reductions in exacerbations and improvements in lung function or quality of life were generally more evident in patients with elevated BEC, suggesting eosinophil levels may help identify those most likely to benefit from biologic therapy.

NEUROLOGY

Frailty and worsening frailty were linked to faster cognitive decline and less favorable cognitive-aging trajectories.

Frailty Linked to Faster Cognitive Decline in Older Adults

A pooled analysis of 19,586 adults from China, England, and the United States found that baseline pre-frailty and frailty, worsening frailty, and greater cumulative frailty burden were associated with faster annual cognitive decline and less favorable cognitive-aging trajectories. Repeated frailty assessments may help identify older adults at greater risk of adverse cognitive aging.

Cochlear implants improved speech perception in adults with prolonged unilateral deafness, supporting individualized candidacy assessment.

Cochlear Implants Improve Speech Perception After Prolonged Deafness

Adults with more than 10 years of unilateral severe-to-profound hearing loss showed meaningful speech-perception gains after cochlear implantation. In a 68-patient multicenter study, 54.5% achieved at least a 20% improvement in word recognition, while mean scores increased by 28.4%. Findings suggest prolonged deafness alone should not exclude patients from CI consideration.

A personalized Mediterranean diet was linked to improved asthma control, body composition, and metabolic markers in obese children.

Mediterranean Diet May Benefit Obese Children With Asthma

An 8-week personalized Mediterranean diet intervention was associated with improved body composition, asthma control, and selected metabolic and inflammatory markers in 37 obese children with asthma. BMI Z-score, waist circumference, and body fat declined, while asthma control improved. Findings are preliminary and require confirmation in larger randomized trials.

Nx3 supports cardiac Z-disk stability and function, while its dysregulation may contribute to heart failure.

Nx3 Titin Isoform Linked to Cardiac Function and Heart Failure

New research identifies Nx3, a small titin isoform, as an important regulator of cardiac muscle structure and performance. Accounting for 20%–25% of titin protein in adult hearts, Nx3 supports Z-disk stability and mechanical function. Its reduced abundance and associated Z-disk disruption in dilated cardiomyopathy suggest a potential role in heart failure pathogenesis.

DENTISTRY

Pterygoid implants showed 97.9% survival and 2.1% failure, supporting their use as a graftless option for posterior maxillary atrophy.

Pterygoid Implants Show 97.9% Survival in Maxillary Atrophy

A systematic review and meta-analysis of 17 studies involving 846 patients and 1,915 pterygoid implants found a pooled implant survival rate of 97.9% and an overall failure rate of 2.1%. Early failures accounted for 0.9%. The findings support pterygoid implants as a graftless option for posterior maxillary atrophy, although evidence certainty remains low to very low.

Epic Bio's EPI-321 Grante...

Epic Bio's EPI-321 Granted FDA Orphan Drug Designation for Innovative FSHD Treatment, Initiates Phase 1/2 Clinical Study