A5043333546- Parkinson's Disease Mechanisms and Treatments
- Chronic Obstructive Pulmonary Disease (COPD) Research
- Asthma and respiratory diseases
- Botulinum Toxin and Related Neurological Disorders
- Cystic Fibrosis Research Advances
- Respiratory Support and Mechanisms
- RNA and protein synthesis mechanisms
- Bacterial Genetics and Biotechnology
- Neurological disorders and treatments
- Hip disorders and treatments
- Influenza Virus Research Studies
- Peptidase Inhibition and Analysis
- Connective tissue disorders research
- Smoking Behavior and Cessation
- Genomics and Rare Diseases
- Sexual Differentiation and Disorders
- RNA regulation and disease
- Advanced biosensing and bioanalysis techniques
- Epilepsy research and treatment
- Pneumonia and Respiratory Infections
- CRISPR and Genetic Engineering
- Respiratory viral infections research
- Autism Spectrum Disorder Research
- Restless Legs Syndrome Research
Johns Hopkins Medicine
2019-2023
Johns Hopkins University
2019-2023
Sunovion (United States)
2019-2023
Vion Pharmaceuticals (United States)
2019-2023
Universidade de São Paulo
2011
Elucidating the functional consequence of molecular defects underlying genetic diseases enables appropriate design therapeutic options. Treatment cystic fibrosis (CF) is an exemplar this paradigm as development CFTR modulator therapies has allowed for targeted and effective treatment individuals harboring specific variants. However, mechanism these drugs limits effectiveness to particular classes variants that allow production protein. Thus, assessment individual imperative proper assignment...
Background: Nausea is common upon initiating dopamine agonists in patients with Parkinson’s disease (PD); however, pretreatment an antiemetic recommended only when apomorphine formulations. Objective: Evaluate the need for prophylactic use during dose optimization of sublingual film (SL-APO). Methods: A post hoc analysis a Phase III study evaluated nausea and vomiting treatment-emergent adverse events PD who underwent SL-APO (10–35 mg; 5-mg increments) to achieve tolerable FULL ON....
Background: Apomorphine sublingual film (SL-APO) and subcutaneous apomorphine (SC-APO) have been used for the treatment of OFF episodes in Parkinson’s disease (PD). No study has prospectively compared efficacy safety these formulations. Objective: To compare SL-APO with SC-APO PD. Methods: An open-label, randomized, crossover assessed versus patients PD (N = 113). Doses were optimized randomly assigned order. dose initiation (10 mg) occurred clinic; further optimization (15–30 mg; 5-mg...
Smoking is a major risk factor for COPD and may impact the efficacy of treatments; however, large proportion patients continue to smoke following diagnosis. This post-hoc analysis pooled data from replicate 12-week, placebo-controlled GEM1 GEM2 studies assessed smoking status on safety glycopyrrolate 15.6 μg twice daily vs placebo in with moderate-to-severe COPD. Data 867 enrolled were grouped by (57% current smokers, 43% ex-smokers). Forced expiratory volume 1 s (FEV1) area under curve 0 12...
Dose optimization of sublingual apomorphine (SL-APO), a dopamine agonist for the treatment OFF episodes in patients with Parkinson's disease (PD), has been performed under clinical supervision trials. SL-APO may be candidate home dosing which would less burdensome patients.
Aim: Evaluate timing of motor improvement with carbidopa/levodopa (CD/LD) and apomorphine sublingual film (SL-APO) in patients Parkinson's disease OFF episodes. Methods: A post hoc pooled analysis from two studies assessed Movement Disorder Society Unified Disease Rating Scale Part III (MDS-UPDRS-III) scores investigator-rated FULL ON. Results: At 15 30 min following the prescribed first daily CD/LD dose, mean improvements MDS-UPDRS-III were -6.7 -16.3, respectively, ON was achieved by 6.5...
Genetic variants that introduce premature termination codons (PTCs) have remained difficult to therapeutically target due lack of protein product. Nonsense mediated mRNA decay (NMD) targets PTC-bearing transcripts reduce the potentially damaging effects truncated proteins. Readthrough compounds been tested on PTC-generating in attempt permit translation through a stop. However, readthrough not proved efficacious clinical setting stable mRNA. Here, we investigate N-terminal cystic fibrosis...
Bronchodilator reversibility has been reported in patients with COPD, although correlations between and treatment response are unclear. The effect of on lung function, health status, dyspnea was assessed moderate-to-severe COPD receiving glycopyrrolate (GLY) 15.6 µg twice daily vs placebo the Glycopyrrolate Effect syMptoms function 1 2 (GEM1 GEM2) replicate, 12-week, placebo-controlled studies.Reversibility defined as a post-bronchodilator increase ≥12% ≥0.200 L FEV1. FEV1 area under curve...
Bronchodilator reversibility occurs in patients with COPD. Pooled analysis of two 12-week, placebo-controlled randomised studies (FLIGHT1 [NCT01727141]; FLIGHT2 [NCT01712516]) assessed the effect bronchodilator on lung function, patient-reported outcomes, and safety 2,043 moderate-to-severe COPD treated indacaterol/glycopyrrolate (IND/GLY) 27.5/15.6 µg twice daily. Reversibility was defined as post-bronchodilator increase forced expiratory volume one second (FEV1) ≥12% ≥0.200 L. Overall,...