Publications by authors named "L R Charnas"
Article Synopsis
- A clinical trial was conducted at Stanford University to test the effectiveness of a 15-day treatment with nirmatrelvir-ritonavir for Post-Acute Sequelae of SARS-CoV-2 infection (PASC) in adults who have experienced moderate to severe symptoms for over 3 months.!* -
- The study involved 155 participants who were randomly assigned to receive either the treatment or a placebo, with the main focus on measuring the severity of six key PASC symptoms after 10 weeks.!* -
- Results indicated no significant improvement in symptom severity between the treatment and placebo groups, suggesting that nirmatrelvir-ritonavir may not effectively alleviate PASC symptoms in the tested population.!*
Article Synopsis
- - The PAX LC trial is a digital and decentralized study aimed at evaluating the effectiveness and safety of nirmatrelvir/ritonavir for long COVID symptoms in 100 adults across the U.S.
- - Participants will be randomly assigned to receive either the medication or a placebo, with data collected through digital diaries and home blood draws to enhance accessibility and engagement.
- - The trial focuses on measuring changes in physical and mental health, alongside exploring biomarkers for long COVID, with a goal of identifying potential treatments for this condition.
Background: Giant axonal neuropathy is a rare, autosomal recessive, pediatric, polysymptomatic, neurodegenerative disorder caused by biallelic loss-of-function variants in , the gene encoding gigaxonin.
Methods: We conducted an intrathecal dose-escalation study of scAAV9/JeT-GAN (a self-complementary adeno-associated virus-based gene therapy containing the transgene) in children with giant axonal neuropathy. Safety was the primary end point.
We evaluated whether whole-body dual-energy X-ray absorptiometry (DXA) measures of lean body mass can be used as biomarkers for disease progression and treatment effects in patients with Duchenne muscular dystrophy. This post hoc analysis utilized data from a randomized, 2-period study of domagrozumab versus placebo in 120 ambulatory boys with DMD. DXA measures of lean body mass were obtained from the whole body (excluding head), arms, legs and appendicular skeleton at baseline and every 16 weeks.
View Article and Find Full Text PDFIntroduction: The North Star Ambulatory Assessment (NSAA) tool is a key instrument for measuring clinical outcomes in patients with Duchenne muscular dystrophy (DMD). To gain a better understanding of the longitudinal utility of the NSAA, we evaluated NSAA data from a phase II trial of 120 patients with DMD treated with domagrozumab or placebo.
Methods: The NSAA exploratory analyses included assessment of individual skills gained/lost, total skills gained/lost, cumulative loss of function, and the impact of transient loss of function due to a temporary disability on NSAA total score (temporary zero score).