AT753 / Astellas - LARVOL DELTA

[VIRTUAL] Retroperitoneal Lymphadenopathy as the First Manifestation of Lymphangioleiomyomatosis (LAM) in Elderly Female (ATS-I 2020) - "Given her lack of symptoms, there was no need to pursue treatment with sirolimus and the patient is being followed clinically with serial PFTs. VEGF-D level was borderline elevated at 753 pg/ml...In our case HRCT subsequently showed very few thin-walled pulmonary cysts. It is also highly unusual to be first diagnosed at this advanced age."

Clinical • Chronic Kidney Disease • Diabetes • Gynecologic Cancers • Metabolic Disorders • Nephrology • Oncology • Rare Diseases • Renal Disease • Solid Tumor • Transplantation • FIGF • Melanosome • VEGFA

Retroperitoneal Lymphadenopathy as the First Manifestation of Lymphangioleiomyomatosis (LAM) in Elderly Female (ATS 2020) - "Given her lack of symptoms, there was no need to pursue treatment with sirolimus and the patient is being followed clinically with serial PFTs. VEGF-D level was borderline elevated at 753 pg/ml.Discussion LAM is often diagnosed in women of child-bearing age and frequently manifests with pulmonary symptoms...In our case HRCT subsequently showed very few thin-walled pulmonary cysts. It is also highly unusual to be first diagnosed at this advanced age."

Clinical • Diabetes • Metabolic Disorders • Nephrology • Oncology • Rare Diseases • Renal Disease • Solid Tumor • FIGF • Melanosome • VEGFA

Porous Cobalt Metal-Organic Frameworks as Active Elements in Battery-Supercapacitor Hybrid Devices. (PubMed, Inorg Chem) - "To further investigate their electrochemical performances, alkaline battery-supercapacitor hybrid (BSH) devices with these materials as positive electrodes and activated carbon (AC) as the negative electrode were fabricated. The results indicate that the PCN//AC BSH devices delivered a maximum energy density of 16.0 Wh kg at a power density of 749 W kg within the voltage range of 0-1.5 V, which are much higher than those of CNFs/PCN//AC devices (12.4 Wh kg at 753 W kg)."

Journal

Audentes Therapeutics reports third quarter 2019 financial results and provides corporate update (Businesswire) - "AT702/AT753/AT751 for Duchenne Muscular Dystrophy (DMD)...IND-enabling dose ranging and toxicology studies underway for AT702; on-track for first quarter 2020 IND submission and plan to initiate a clinical study in the second quarter of 2020....AT753 exon 53 targeting oligonucleotide sequence selected; manufacturing underway to support IND-enabling preclinical studies to be initiated this quarter....AT751 exon 51 targeting oligonucleotide screening underway; plan to initiate IND-enabling preclinical studies in the first quarter of 2020."

IND • New trial • Preclinical