Blog
Risdiplam for Prenatal Therapy of Spinal Muscular Atrophy
THE NEW ENGLAND JOURNAL OF MEDICINE Risdiplam for Prenatal Therapy of Spinal Muscular Atrophy Published February 19, 2025 | DOI: 10.1056/NEJMc2300802 | Copyright © 2025 Abstract The alternate splicing small molecule risdiplam, administered to a fetus with spinal...
Promizing CD206/CD209/IL6 decrease in TME by ancentrally active antagonist
ASCO 2025: Promizing CD206/CD209/IL6 decrease in TME by ancentrally active antagonist of C/EBPbleading to positive trends in PFS and OS in GBM patients; based on solid non-clinical evidence (PI/II: NCT04478279) Use of lucicebtide (ST101) in glioblastoma patients by...
A very interesting link between glycosylation and exhaustion described by Azevedo et coll. I, Cancer Immunol Res.
A very interesting link between glycosylation and exhaustion described by Azevedo et coll. I, Cancer Immunol Res. Mgat5 played a prominent role in T-cell exhaustion. In culture-based assays and tumor studies, Mgat5 deletion in CD8+ T cells resulted in improved cancer...
Revach OY, et coll. showed the importance of CD38 as new additional player in CD8 defense
Revach OY, et coll. showed the importance of CD38 as new additional player in CD8 defense restauration in melanoma PDX ex vivo. Read this oustsanding work in Cell Rep Med. 2025 Jun 27;6(7):102210 CD38 as a Therapeutic Target in Melanoma CD38 is upregulated in...
A recent multicenter, randomized, double-blind, placebo-controlled study in 194 (full set analysis)
A recent multicenter, randomized, double-blind, placebo-controlled study in 194 (full set analysis. Generalized anxiety disorder-patients showed a significant decrease of -5 points in Hamilton Anxiety Rating Scale at day 28 with a single dose of 100 microgram of...
A newly identified macrophage manager ZEB2
A newly identified macrophage manager ZEB2: still known as pivotal in early fetal development and cancer progression by driving the epithelial-to-mesenchymal transition. Now proven as a master switch controlling the tumor-associated macrophage program Fadi Sheban ,...
In 2023, the FDA granted approval to 13 new oncology drugs, constituting ~24% of the total approvals for the year
First-in-Class Therapies: AstraZeneca’s Capivasertib (Truqap): first AKT inhibitor approved for hormone-receptor-positive, HER2-negative metastatic breast cancer. Its approval alongside a companion diagnostic for PIK3CA, AKT1, or PTEN alterations; precision medicine...
Plasmatic IL8 as COPD biomarker response to PDE treatments? A very promising tool!
“The study by Govoni and colleagues (Govoni M, Bassi M, Santoro D, Donegan S, Singh D. Serum interleukin-8 as a determinant of response to PDE4 inhibition in chronic obstructive pulmonary disease. Am J Respir Crit Care Med 2023; 208:559–569.) opens a new field of...
Started in oncology and opening a new era in CNS and beyond!
Many CNS diseases lack curative or disease-modifying treatments and represent a growing burden to healthcare systems and societies worldwide. These diseases are often multifactorial and complex in nature, with significant individual variability in the underlying...