A5006699282- Asthma and respiratory diseases
- Inhalation and Respiratory Drug Delivery
- Neurogenetic and Muscular Disorders Research
- Respiratory and Cough-Related Research
- RNA modifications and cancer
- Chronic Obstructive Pulmonary Disease (COPD) Research
- Pharmaceutical studies and practices
- RNA Research and Splicing
- Pharmacological Effects and Assays
- Health Systems, Economic Evaluations, Quality of Life
- Musculoskeletal pain and rehabilitation
- Muscle Physiology and Disorders
- Biomedical Ethics and Regulation
- Spine and Intervertebral Disc Pathology
- Respiratory Support and Mechanisms
- Family and Disability Support Research
- Child Nutrition and Feeding Issues
- Cardiovascular and exercise physiology
- Science, Research, and Medicine
- Cystic Fibrosis Research Advances
- Immunodeficiency and Autoimmune Disorders
- Tissue Engineering and Regenerative Medicine
- Mechanical Circulatory Support Devices
- Hormonal Regulation and Hypertension
- Cardiomyopathy and Myosin Studies
Roche (Switzerland)
2024
Roche (United Kingdom)
2018-2024
Roche (Bosnia-Herzegovina)
2020-2023
Mundipharma (United Kingdom)
2011-2018
St. Cloud Orthopedic Associates
2018
Abstract Risdiplam is an oral, survival of motor neuron 2 ( SMN2 ) pre-mRNA splicing modifier approved for the treatment spinal muscular atrophy (SMA). SUNFISH (NCT02908685) Part 2, a Phase 3, randomized, double-blind, placebo-controlled study, investigated efficacy and safety risdiplam in type non‑ambulant 3 SMA. The primary endpoint was met: significantly greater change from baseline 32-item Motor Function Measure (MFM32) total score observed with compared placebo at month 12. After 12...
Abstract Background and purpose Spinal muscular atrophy (SMA) is caused by reduced levels of survival motor neuron (SMN) protein due to deletions and/or mutations in the SMN1 gene. Risdiplam an orally administered molecule that modifies SMN2 pre‐mRNA splicing increase functional SMN protein. Methods SUNFISH Part 1 was a dose‐finding study conducted 51 individuals with types 2 3 SMA aged 2–25 years. A dose‐escalation method used identify appropriate dose for subsequent pivotal 2. Individuals...
Despite efforts to increase diversity in neuroscience trials, racial and ethnic minority groups remain underrepresented. Disparities clinical trial participation could reflect unequal opportunities participate may contribute decreased generalizability of findings failure identify important differences efficacy safety outcomes. We retrospectively reviewed the F. Hoffmann-La Roche database for global, multicenter, trials from February 2016 2021 summarized stratified race ethnicity...
The potent inhaled corticosteroid, fluticasone propionate (fluticasone), and the long-acting β2-agonist with a rapid onset of action, formoterol fumarate (formoterol), have now been combined in single aerosol inhaler, fluticasone/formoterol (flutiform). This study investigated efficacy safety combination therapy compared its individual components administered concurrently via two separate inhalers.Patients ≥ 12 years (N = 210) mild to moderate-severe persistent, reversible asthma were evenly...
The inhaled corticosteroid, fluticasone propionate (fluticasone), and the long-acting beta2-agonist, formoterol fumarate (formoterol), have been combined in a single aerosol inhaler (fluticasone/formoterol). In randomized, open-label study, fluticasone/formoterol showed similar efficacy to fluticasone/salmeterol after 12 weeks of treatment. This post-hoc analysis compared onset bronchodilation with two treatments. Adults mild-to-moderate-severe persistent asthma were randomized (100/10 or...
A primary goal of asthma management is the reduction exacerbation risk. We assessed occurrence oral corticosteroid-requiring exacerbations (OCS exacerbations) with long-term fluticasone/formoterol therapy, and compared it similar events reported other inhaled corticosteroid/long acting β2-agonist (ICS/LABA) combinations.
Background: The efficacy and safety of fluticasone propionate/formoterol fumarate pressurized metered-dose inhaler (pMDI) (fluticasone/formoterol; Flutiform ® ; 100/10 µg b.i.d.) was compared with propionate (Flixotide Evohaler pMDI; 100 fluticasone/salmeterol (Seretide 100/50 in a pediatric asthma population (EudraCT number: 2010-024635-16). Methods: A double-blind, double-dummy, parallel group, multicenter study. Patients, aged 5–<12 years persistent ⩾ 6 months forced expiratory volume...
Objectives: The present study was conducted to assess the efficacy, safety and tolerability of fluticasone propionate/formoterol fumarate combination therapy (FP/FORM; Flutiform®) compared with propionate/salmeterol xinafoate (FP/SAL; Seretide® Evohaler®) in children asthma. Methods: This an open-label, randomized, controlled, phase III trial extension. Patients aged 4–12 years reversible asthma [% predicted forced expiratory volume 1 second (FEV ) 60–100%; documented reversibility ⩾15% FEV...
Despite extensive use of inhaled corticosteroid/long-acting β2-agonist combinations in asthma, limited data evaluating dose-response for this combination class are available. The benefits dose escalation and nature patient subgroups likely to benefit thus ill-defined.In randomised, double-blind, 8-week study the effects two levels (100/10 500/20 μg b.i.d.) a fixed fluticasone/formoterol (flutiform(®)) were compared 309 patients. Treatment upon spirometric symptom-based endpoints examined...
Aims The aim of this study was to determine if positive Waddell signs were related patients’ demographics or perception their quality life. Patients and Methods This prospective cross-sectional included 479 adult patients with back pain from a university spine centre. Each completed SF-12 Oswestry Disability Index (ODI) questionnaires underwent standard spinal examinations elicit signs. relationship between age, gender, ODI, Mental Component Score (MCS), Physical (PCS) scores determined....
Abstract: Inhaled corticosteroid/long-acting β 2 -agonist combination therapy is recommended in chronic obstructive pulmonary disease (COPD) patients at high risk of exacerbations. The EFFECT (Efficacy Fluticasone propionate/FormotErol COPD Treatment) trial a Phase III, 52-week, randomized, double-blind study to evaluate the efficacy and safety two doses fluticasone propionate/formoterol compared formoterol monotherapy with FEV 1 ≤50% predicted history primary endpoint annualized rate...
Objective: To describe the longer-term safety profile of risdiplam (RG7916) in individuals who have participated FIREFISH, SUNFISH, JEWELFISH and RAINBOWFISH studies. Background: Spinal muscular atrophy (SMA) is a severe, progressive neuromuscular disease caused by reduced levels survival motor neuron (SMN) protein due to deletions and/or mutations SMN1 gene. A second gene, SMN2, produces only low functional SMN protein. Risdiplam an orally administered, centrally peripherally distributed...
<h3>Background</h3> Fluticasone propionate (FP) and formoterol (FORM) have been combined in a single inhaler (FP/FORM;<b><i>flutiform</i></b><sup>®</sup>) for the treatment of adolescents adults with asthma. This study assessed efficacy safety FP/FORM paediatric asthma patients. <h3>Methods</h3> A total 512 patients aged 5 to <12yrs were randomised 1:1:1 12 weeks either (100/10 µg BID), FP (100 BID) or fluticasone propionate/salmeterol (FP/SAL) (100/50 double-blind, parallel group,...
Facioscapulohumeral muscular dystrophy (FSHD) is a genetic disorder characterized by progressive muscle weakness leading to permanent disability. There are no curative treatments, however, there several upcoming clinical trials testing new therapies in FSHD.
Social anxiety disorder (SAD) affects up to 1 in 8 individuals over their lifetime and is characterized by an intense fear of social situations where there may be exposure unfamiliar people or possible scrutiny. The analysis media data rather than traditional methods (interviews focus groups) can provide a unique opportunity understand the lived experience with SAD, for whom interacting strangers challenging. This retrospective observational study reviewed published literature from PubMed...