Radboud University Medical Center

Philikos Initiates Phase 1/2 Trial of T-Guard for Diffuse Cutaneous Systemic Sclerosis

2 days ago

• Philikos has enrolled the first patient in a Phase 1/2 trial evaluating T-Guard for diffuse cutaneous systemic sclerosis (dcSSc), a severe autoimmune disorder with limited treatment options. • The open-label study will assess safety and preliminary efficacy in 12 early-stage dcSSc patients whose disease remains refractory despite prior immunosuppressive therapy. • T-Guard, administered as four infusions over one week, aims to provide a safer alternative to hematopoietic stem cell transplantation by selectively depleting disease-associated T cells and NK cells.

PacBio and Radboud UMC Unveil Groundbreaking Study Results Using HiFi Long-Read Sequencing

4 months ago

A recent study published in the American Journal of Human Genetics by PacBio and Radboud University Medical Center highlights the transformative potential of HiFi long-read sequencing technology in diagnosing rare diseases. The study demonstrated a 93% success rate in identifying pathogenic variants, offering a more comprehensive and cost-effective diagnostic approach. This collaboration is paving the way for the integration of HiFi sequencing into clinical practice, with significant advancements in sample processing and operational efficiency.

Trametinib Shows Promise in Treating Severe Hypertrophic Cardiomyopathy in Children

4 months ago

• A recent study published in JACC: Basic to Translational Science indicates that trametinib reduces mortality and morbidity in children with severe hypertrophic cardiomyopathy (HCM). • The study focused on children with HCM caused by pathogenic variants in the RAS/MAPK pathway, highlighting the potential for personalized treatment approaches. • Results showed a significant reduction in death, cardiac transplantation, or the need for cardiac surgery in children treated with trametinib compared to standard care. • While dermatologic and mucous membrane side effects were common, no life-threatening adverse events were observed, suggesting manageable safety profiles.

RH5.1/Matrix-M Malaria Vaccine Shows Promise as Blood-Stage Defense

6 months ago

• A Phase IIb trial of the RH5.1/Matrix-M vaccine shows 55% efficacy in preventing clinical malaria over six months in young children. • The vaccine also demonstrated over 80% efficacy against high levels of malaria parasites, suggesting potential in preventing severe cases. • RH5.1/Matrix-M targets the blood-stage of malaria, complementing existing vaccines that target the liver-stage of the parasite. • The RH5.1/Matrix-M vaccine was well-tolerated, with mild adverse events, positioning it as a potential second line of defense against malaria.

Immunotherapy Shows Promise in Prostate Cancer with MMRd Mutation

8 months ago

• Men with prostate cancer and the MMRd mutation experienced significantly extended progression-free survival with dual immunotherapy. • The disease control rate at six months in MMRd tumors was 81%, substantially higher than other mutation types. • Median progression-free survival in the MMRd group reached 32.7 months, a notable improvement compared to other groups. • These findings suggest that immunotherapy could be a beneficial treatment option for a subset of prostate cancer patients with specific genetic profiles.

PLEASE Trial Assesses Prolonged Antibiotic Treatment for Persistent Lyme Symptoms

11 years ago

• The Persistent Lyme Empiric Antibiotic Study Europe (PLEASE) investigates optimal treatment for borreliosis-attributed persistent symptoms. • The study is a randomized, double-blind, placebo-controlled trial comparing short-term versus long-term antibiotic regimens. • Researchers aim to determine if prolonged antibiotic treatment improves patient outcomes compared to standard short-term therapy. • The trial includes patients with persistent pain, fatigue, and cognitive issues following Lyme disease, evaluating physical and mental health.