by Justin Jackson, Medical Xpress Taxonomy of ASO-amenable variants. Two-step logic for classifying the amenability of genetic variants to splice modulation rescue (probably, possibly or unlikely). First, each variant is evaluated for its damaging impact on either canonical splicing or protein-coding function, on the basis of SpliceAI, MaxEntScan, LaBranchoR and REVEL. Second, each variant is...
Liam Davenport July 13, 2023 An unapproved gene therapy for Leber hereditary optic neuropathy (LHON) led to a marked improvement in the eyesight of patients with a severe, progressive form of the disease who received the therapy as part of an early access program. Results of a study of more than 60 patients who received lenadogene nolparvovec...
by Universita Cattolica del Sacro Cuore Credit: CC0 Public Domain Neuroscientists from the Faculty of Medicine of the Catholic University, Rome Campus, and the A. Gemelli IRCCS Polyclinic Foundation have found that intensive exercise could slow the course of Parkinson’s disease. The finding could pave the way for new non-drug approaches. The study, “Intensive exercise ameliorates motor...
By STACY LIBERATORE FOR DAILYMAIL.COM UPDATED: 12:17 EDT, 14 July 2023 Harvard scientists believe they are one step closer to a fountain of youth pill that can de-age people. The team discovered six chemical cocktails that reversed the aging process in human and mice skin cells ‘by several years’. Dr David Sinclair, a molecular biologist at Harvard...
by Impact Journals LLC Reprogramming small molecule cocktails restore NCC alterations in senescent cells. (A) Chemical structures of small molecules of basal cocktails used to generate induced pluripotent stem cells (iPSCs) from mouse (left) or human (right) somatic cells. (B) Correlation heatmaps showing eGFP and mCherry colocalization in human senescent fibroblasts demonstrate the effects of...
Miriam E. Tucker June 24, 2023 SAN DIEGO – Orforglipron, Eli Lilly’s investigational oral daily nonpeptide glucagon-like peptide-1 (GLP-1) agonist that can be taken with or without food, appears comparable with other injectable and oral agents in the class for treating obesity and type 2 diabetes, suggest two new phase 2 studies. Currently approved GLP-1 agonists for type...
Miriam E. Tucker July 03, 2023 SAN DIEGO — An investigational allogeneic stem cell–derived pancreatic islet cell replacement therapy (VX-880, Vertex Pharmaceuticals) continues to show promise as a treatment for type 1 diabetes, according to the latest data, from six patients thus far. Two of the six are insulin-independent beyond 1 year after receiving the VX-880 infusions,...
Mitchel L. Zoler, PhD June 26, 2023 SAN DIEGO — New designer molecules that target weight loss via multiple mechanisms continue to raise the bar of how many pounds people with overweight or obesity can lose. Retatrutide (Eli Lilly), an investigational agent that combines agonism to three key hormones that influence eating and metabolism into a single...
Marcus A. Banks July 11, 2023 The US Food and Drug Administration has approved a new catheter designed to treat urinary tract symptoms of an enlarged prostate, also known as benign prostate hyperplasia (BPH). Designed and marketed by Urotronic (Plymouth, Minnesota), the Optilume BPH Catheter System employs mechanical dilation to relieve obstruction of the prostate and then...
Yoni Freedhoff, MD July 11, 2023 Are there medical conditions other than obesity where physicians, if even inadvertently, regularly sabotage their patients’ efforts at managing them? Because for patients with obesity, their physicians, sometimes even before they walk into the examining room to meet them, do and say things that may quash their weight loss efforts. No doubt this list will...
