A5027964069- Neurogenetic and Muscular Disorders Research
- Peripheral Artery Disease Management
- RNA modifications and cancer
- Psoriasis: Treatment and Pathogenesis
- Autoimmune Bullous Skin Diseases
- Cerebrovascular and Carotid Artery Diseases
- CRISPR and Genetic Engineering
- Health Systems, Economic Evaluations, Quality of Life
- Pain Mechanisms and Treatments
- Antiplatelet Therapy and Cardiovascular Diseases
- Botulinum Toxin and Related Neurological Disorders
- Congenital Anomalies and Fetal Surgery
- Pharmaceutical studies and practices
- Multiple Sclerosis Research Studies
- Diabetic Foot Ulcer Assessment and Management
- Peripheral Neuropathies and Disorders
- Schizophrenia research and treatment
- Systemic Sclerosis and Related Diseases
- Venous Thromboembolism Diagnosis and Management
- Rheumatoid Arthritis Research and Therapies
- Pain Management and Treatment
- Fibromyalgia and Chronic Fatigue Syndrome Research
- Bipolar Disorder and Treatment
- Genetics and Neurodevelopmental Disorders
- Diagnosis and Treatment of Venous Diseases
Novartis (United States)
2021-2023
Statistical Research (United States)
2022-2023
Novartis (Germany)
2021-2022
Novartis Gene Therapies, Inc. (United States)
2021-2022
Meridian Clinical Research
2018-2021
Baxalta (United States)
2019-2020
University of Colorado Denver
2020
UCB Pharma (United States)
2019
Apex School of Theology
2019
Montreal Neurological Institute and Hospital
2018
Abstract SPR1NT ( NCT03505099 ) was a Phase III, multicenter, single-arm study to investigate the efficacy and safety of onasemnogene abeparvovec for presymptomatic children with biallelic SMN1 mutations treated at ≤6 weeks life. Here, we report final results 14 two copies SMN2 , expected develop spinal muscular atrophy (SMA) type 1. Efficacy compared matched Pediatric Neuromuscular Clinical Research natural-history cohort n = 23). All enrolled infants sat independently ≥30 seconds any visit...
Most children with biallelic SMN1 deletions and three SMN2 copies develop spinal muscular atrophy (SMA) type 2. SPR1NT ( NCT03505099 ), a Phase III, multicenter, single-arm trial, investigated the efficacy safety of onasemnogene abeparvovec for presymptomatic mutations treated within six postnatal weeks. Of 15 before symptom onset, all stood independently 24 months (P < 0.0001; 14 normal developmental window), walked 11 window). All survived without permanent ventilation at months; ten (67%)...
<h3>Objective</h3> To assess dose-response effects of the anti-CD20 monoclonal antibody ofatumumab on efficacy and safety outcomes in a phase 2b double-blind study relapsing forms multiple sclerosis (RMS). <h3>Methods</h3> Patients (n = 232) were randomized to 3, 30, or 60 mg every 12 weeks, 4 placebo for 24-week treatment period, with primary endpoint cumulative number new gadolinium-enhancing lesions (per brain MRI) at week 12. Relapses safety/tolerability assessed, CD19+ peripheral blood...
Objective: To evaluate Positive and Negative Syndrome Scale (PANSS) categorical response rates, time course of response, symptom subdomains with the combination oral agent KarXT (xanomeline-trospium) in treatment schizophrenia.Methods: Post hoc analysis was conducted for EMERGENT-1 (NCT03697252), a 5-week, inpatient, placebo-controlled, phase 2 study acute psychosis patients who met DSM-5 criteria schizophrenia. The between September 2018 August 2019. Categorical thresholds used were PANSS...
Spinal muscular atrophy (SMA) is a neuromuscular disorder arising from biallelic non-functional survival motor neuron 1 (SMN1) genes with variable copies of partially functional SMN2 gene. Intrathecal onasemnogene abeparvovec administration, at fixed, low doses, may enable treatment heavier patients ineligible for weight-based intravenous dosing.STRONG (NCT03381729) assessed the safety/tolerability and efficacy intrathecal sitting, nonambulatory SMA patients.Sitting, (biallelic SMN1 loss,...
Preliminary evidence suggests that vagus nerve stimulation (VNS) may have some benefit in patients with rheumatoid arthritis (RA); however, prior studies been small and/or uncontrolled; this study aimed to address gap.This randomized, double-blind, sham-controlled trial enrolled aged 18 75 years active RA who had failed conventional synthetic disease-modifying antirheumatic drugs (DMARDs) and were naïve biologic targeted DMARDs. All received an auricular stimulator randomized 1:1 or sham....
Abstract Background Gabapentin enacarbil ( GE n), a transported prodrug of gabapentin, provides sustained, dose‐proportional gabapentin exposure. The purpose this study was to investigate the dose response n select optimal dose(s) for clinical use in subsequent diabetic peripheral neuropathy DPN ) trials. Methods This multicenter, randomized, double‐blind, double‐dummy, parallel group, placebo‐controlled trial with duration approximately 20 weeks Clinicaltrials.gov database, Identifier ! NCT...
Current guidelines recommend aggressive management of hypertension. Recent evidence suggested potential harm with low blood pressure targets in patients peripheral artery disease. We investigated the association a history hypertension and office systolic (SBP) major adverse cardiovascular events (MACEs) limb (MALEs).The EUCLID trial (Examining Use Ticagrelor Peripheral Artery Disease) included 13 885 participants symptomatic disease; median follow-up was 30 months. Cox proportional hazards...
In patients with symptomatic peripheral artery disease (PAD), the impact of chronic kidney (CKD) on major adverse cardiovascular events has not been fully evaluated. The Examining Use Ticagrelor PAD (EUCLID) trial randomized 13,885 to ticagrelor 90 mg twice daily or clopidogrel 75 daily. This post hoc analysis compared incidence primary composite endpoint (cardiovascular death, myocardial infarction (MI), ischemic stroke) in CKD (eGFR < 60 mL/min/1.73 m 2 ) those without ⩾ ). safety was...
This study evaluates safety and efficacy of onasemnogene abeparvovec-xioi (formerly AVXS-101), a survival motor neuron gene (SMN)–replacement therapy, in presymptomatic SMA patients.
To compare the efficacy of high-dose (3,600 mg/day) vs low-dose (1,200 oral gabapentin enacarbil (GEn) on pain intensity in adults with postherpetic neuralgia (PHN) and a history inadequate response to ≥1,800 mg/day gabapentin. Multicenter, randomized, double-blind, crossover study (NCT00617461). Thirty-five outpatient centers Germany United States. Subjects aged ≥18 years diagnosis PHN. During 2-week baseline period, subjects received open-label treatment 1,800 who had mean 24-hour average...
Evaluate the safety/efficacy of onasemnogene abeparvovec (formerly AVXS-101) in presymptomatic SMA patients with 2 copies survival motor neuron gene (SMN2).
CATIE-AD was a multicenter effectiveness trial of atypical antipsychotics in patients with agitation and psychosis associated AD who resided the community. The study enrolled 421 participants. In this paper we present discuss baseline characteristics participants (demographics, cognitive, behavioral, functional assessments), caregivers (demographics caregiver burden) settings at randomization. Those suffered from wide range cognitive impairment, were medically complex experienced acute...
To assess the safety/tolerability, optimal dose, and efficacy of AVXS-101 IT in sitting but non-ambulatory patients with spinal muscular atrophy (SMA).
Evaluate the safety/efficacy of onasemnogene abeparvovec (formerly AVXS-101) in presymptomatic SMA patients with 3 copies survival motor neuron 2 gene (SMN2).
Abstract not available.