Download full-text PDF |
Source |
|---|---|
| http://dx.doi.org/10.1016/j.jaad.2017.08.056 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
Safety and Vision Outcomes Following Gene Therapy for Bietti Crystalline Dystrophy: A Nonrandomized Clinical Trial.
JAMA Ophthalmol
January 2025
Xiamen Eye Center of Xiamen University, Xiamen, Fujian, China.
Importance: Bietti crystalline dystrophy (BCD) is a severe genetic retinopathy caused by variants in the CYP4V2 gene. Currently, there is no approved treatment for BCD.
Objective: To evaluate safety and vision outcomes following gene therapy with adeno-associated virus (AAV) encoding CYP4V2 (rAAV-hCYP4V2, NGGT001 [Next Generation Gene Therapeutics]).
Phase 2 Open-Label Trial of Brentuximab Vedotin with Pembrolizumab in PD-1 Pretreated Metastatic Non-Small Cell Lung Cancer and Metastatic Cutaneous Melanoma.
Clin Cancer Res
December 2024
Baylor University Medical Center, Dallast, Texas, United States.
Purpose: Brentuximab vedotin (BV) is hypothesized to selectively deplete T regulatory cells (Tregs) that express CD30 and re-sensitize tumors to anti-(PD-1) therapy. This study evaluated responses to BV+pembrolizumab post PD-1 and explored corresponding biomarkers.
Methods: 55 patients with metastatic non-small cell lung cancer (NSCLC) and 58 with metastatic cutaneous melanoma received ≥1 dose of BV+pembrolizumab.
Dose‐related modulation of the synaptic proteome after short‐term treatment with XPro1595 for Alzheimer’s disease.
Alzheimers Dement
December 2024
INmune Bio, Boca Raton, FL, USA
Background: XPro1595 (XPro) is a brain‐penetrant, recombinant protein variant of human tumor necrosis factor (TNF) rationally designed to selectively neutralize only the soluble, pro‐inflammatory form of the cytokine (solTNF). An unbiased proteomic analysis of CSF samples from an open‐label, phase‐1b study (NCT03943264) in patients with Alzheimer’s disease (AD) was conducted to assess for pharmacodynamic activity and disease‐specific target engagement.
Method: Patients with AD (n = 20) were treated for 12‐weeks with one of three doses of XPro: 0.
Delaying symptom onset in Dominantly Inherited Alzheimer’s Disease: Long‐term gantenerumab treatment results from the DIAN‐TU trial.
Alzheimers Dement
December 2024
F. Hoffmann‐La Roche Ltd, Basel, Switzerland
Background: Amyloid‐plaque removal by monoclonal antibody therapies slows progression in symptomatic Alzheimer’s disease (AD), but effects on preventing the onset of symptoms and dementia in asymptomatic people with amyloid plaques are unknown. We report the final primary and secondary outcomes of the Dominantly Inherited Alzheimer Network Trials Unit (DIAN‐TU) trial to evaluate amyloid‐plaque removal in delaying disease progression, including symptom onset, in symptomatic and asymptomatic dominantly inherited Alzheimer’s disease (DIAD) individuals treated for up to a decade.
Method: This double‐blind, phase 2/3 trial (2012‐2019), followed by open‐label extension (OLE), investigated varying gantenerumab doses up to 1500 mg subcutaneous q2 weeks [NCT01760005].
Safety and Tolerability of Wharton's Jelly-Derived Mesenchymal Stem Cells for Patients With Duchenne Muscular Dystrophy: A Phase 1 Clinical Study.
J Clin Neurol
January 2025
Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea.
Background And Purpose: This study was an open-label, dose-escalation, phase 1 clinical trial to determine the safety and dose of EN001 for patients with Duchenne muscular dystrophy (DMD). EN001, developed by ENCell, are allogeneic early-passage Wharton's jelly-derived mesenchymal stem cells that originate at the umbilical cord, with preclinical studies demonstrating their high therapeutic efficacy for DMD.
Methods: This phase 1 clinical trial explored the safety and tolerability of EN001 as a potential treatment option for patients with DMD.
Want AI Summaries of new PubMed Abstracts delivered to your In-box?
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!