A5014387622- Growth Hormone and Insulin-like Growth Factors
- Pituitary Gland Disorders and Treatments
- Diet and metabolism studies
- Ocular Surface and Contact Lens
- Childhood Cancer Survivors' Quality of Life
- Animal Vocal Communication and Behavior
- Neural dynamics and brain function
- Glaucoma and retinal disorders
- Cancer, Hypoxia, and Metabolism
- Hormonal Regulation and Hypertension
- Adrenal Hormones and Disorders
- Cannabis and Cannabinoid Research
- Lipoproteins and Cardiovascular Health
- Visual perception and processing mechanisms
- Thyroid and Parathyroid Surgery
- Birth, Development, and Health
- Neurotransmitter Receptor Influence on Behavior
- Hearing, Cochlea, Tinnitus, Genetics
- Sexual Differentiation and Disorders
- Corneal Surgery and Treatments
- Sepsis Diagnosis and Treatment
- Neuroscience and Neuropharmacology Research
- Hyperglycemia and glycemic control in critically ill and hospitalized patients
- Allergic Rhinitis and Sensitization
- Autism Spectrum Disorder Research
Ascendis Pharma (Denmark)
2017-2022
Albert Einstein College of Medicine
2015-2020
Children's Hospital of Los Angeles
2019
Clinical Research Services
2017-2019
University of Maryland, Baltimore
2012
To compare the efficacy, safety, and tolerability of waterfree cyclosporine formulation (CyclASol) at 2 concentrations (0.1% 0.05% [CsA]) to vehicle when applied twice daily for 16 weeks in patients with dry eye disease (DED). An open-label Restasis (Allergan, Irvine, CA) arm was included allow a direct comparison an approved therapy.An exploratory phase II, multicenter, randomized, vehicle-controlled clinical trial, double-masked between CyclASol comparator.Two hundred seven eligible...
For children with growth hormone deficiency (GHD), treatment burden daily somatropin injections [human (hGH)] is high, which may lead to poor adherence and suboptimal overall outcomes. Lonapegsomatropin (TransCon hGH) an investigational long-acting, once-weekly prodrug for the of GHD.The objective this study was evaluate efficacy safety lonapegsomatropin vs somatropin.The heiGHt trial a randomized, open-label, active-controlled, 52-week Phase 3 (NCT02781727).This took place at 73 sites...
Purpose: Evaporation of the tear film is heavily discussed as one core reason for dry eye disease (DED). Subsequently, new artificial products are developed that specifically target this pathomechanism. Perfluorohexyloctane (F6H8, NovaTears®) from family semifluorinated alkanes a novel substance has been approved medical device, nonblurring wetting agent ocular surface. Methods: Thirty patients with hyperevaporative received F6H8 during prospective, multicenter, observational 6-week study....
Meibomian gland disease is generally accepted as the leading cause for evaporative dry eye (DED). In a previous study, perfluorohexyloctane, semifluorinated alkane, has been demonstrated to significantly increase tear film breakup time and reduce corneal fluorescein staining in patients with DED, thereby vastly reducing eye-related symptoms. This study was set up evaluate perfluorohexyloctane larger population of dysfunction.Seventy-two associated received 1 drop 4 times daily during an...
Abstract Context: TransCon Growth Hormone (GH) (Ascendis Pharma) is a long-acting recombinant sustained-release human GH prodrug in development for children with deficiency (GHD). Objective: To compare the pharmacokinetics, pharmacodynamics, safety, and efficacy of weekly to that daily prepubertal GHD. Design: Randomized, open-label, active-controlled study three doses versus Genotropin (Pfizer). Setting: Thirty-eight centers 14 European countries Egypt. Patients: Prepubertal male female...
Abstract Purpose The objectives of the ongoing, Phase 3, open-label extension trial enliGHten are to assess long-term safety and efficacy weekly administered long-acting growth hormone lonapegsomatropin in children with deficiency. Methods Eligible subjects completing a prior 3 parent (heiGHt or fliGHt) were invited participate. All treated lonapegsomatropin. Subjects United States switched TransCon hGH Auto-Injector when available. Endpoints safety, annualized height velocity,...
TransCon growth hormone is a sustained-release human prodrug under development in which unmodified transiently linked to carrier molecule. It intended as an alternative daily the treatment of deficiency. This was multi-center, randomized, open-label, active-controlled trial designed compare safety (including tolerability and immunogenicity), pharmacokinetics pharmacodynamics three doses weekly GH (Omnitrope). Thirty-seven adult males females diagnosed with deficiency stable on replacement...
Objective: The objectives of this study were to evaluate safety (primary) and clinical efficacy (secondary) the humanized monoclonal anti-L-selectin antibody aselizumab in severely injured patients. Design: Prospective phase II, parallel group, double-blind, randomized, placebo-controlled trial. Setting: Fourteen medical intensive care units or trauma level I centers Belgium, Germany, Poland. Patients: Eighty-four patients with a sustained due blunt penetrating injury total Injury Severity...
The midbrain map of auditory space commands sound-orienting responses in barn owls. Owls precisely localize sounds frontal but underestimate the direction peripheral sound sources. This bias for central locations was proposed to be adaptive decreased reliability periphery sensory cues used localization by owl. Understanding neural pathway supporting this biased behavior provides a means address how motor are implemented neurons. Here we find that input is weighted its premotor neurons...
A major cue to infer sound direction is the difference in arrival time of at left and right ears, called interaural (ITD). The neural coding ITD its similarity across species have been strongly debated. In barn owl, an auditory specialist relying on localization capture prey, ITDs within physiological range determined by head width are topographically represented each frequency. topographic representation suggests that may be inferred from location maximal activity map. Such topographical...
Abstract While a topographic map of auditory space exists in the vertebrate midbrain, it is absent forebrain. Yet, both brain regions are implicated sound localization. The heterogeneous spatial tuning adjacent sites forebrain compared to midbrain reflects different underlying circuitries, which expected affect correlation structure, i.e., signal (similarity tuning) and noise (trial-by-trial variability) correlations. Recent studies have drawn attention impact response correlations on...
e13551^ Background: Most of the protein kinase inhibitors (PKIs) have very challenging solubility properties and many products are suffering from pH dependent absorption, food effect high variability leading to unmet medical needs [Di Gion et al.; Clin Pharmacokinet. 2011 Sep 50(9):551-603.]. We earlier shown outstanding in vitro results with 8 different PKIs formulated RightSize hybrid nanoparticles. The formulation improvments were verified a preclinical dog model using Nilotinib [Jesson...
Neonatal vocalization is structurally altered in mouse models of autism spectrum disorder (ASD). Our published data showed that pup vocalization, under conditions maternal separation, contains sequences whose alterations a genetic model ASD impair social communication between pups and mothers. We describe details method which reveals the statistical structure call are functionally critical for optimal care. Entropy analysis determines degree non-random sequencing. A Markov actual used by...
TransCon Growth Hormone in the Treatment of Pediatric Deficiency: Results Phase 3 heiGHt TrialBackground is a sustained-release recombinant human growth hormone (hGH; somatropin) prodrug development as long-acting GH (LAGH) for children with deficiency (GHD). In its form, hGH inactive transiently bound to carrier via linker. Upon injection and triggered by physiological pH temperature, unmodified sustainably released thus designed maintain same mode-of-action distribution daily replacement...
In this graphical review we highlight key milestones in the discovery and development of CRISPR cas9 technology, as well illustrate mechanisms cas9:sgRNA target binding DNA repair.
Abstract Background: Once-weekly TransCon hGH (lonapegsomatropin) is an investigational long-acting prodrug of somatropin in development for GHD. In the pivotal 52-week phase 3 heiGHt trial, lonapegsomatropin demonstrated superior annualized height velocity (AHV) compared to same weekly dose daily treatment-naïve children with 26-week fliGHt switch from provided continued growth and maintained a good safety profile. Methods: Results are reported subjects who into open-label long-term...
Abstract The midbrain map of auditory space commands sound-orienting responses in barn owls. Owls precisely localize sounds frontal but underestimate the direction peripheral sound sources. This bias for central locations was proposed to be adaptive decreased reliability periphery sensory cues used localization by owl. Understanding neural pathway supporting this biased behavior provides a means address how motor are implemented neurons. Here we find that input is weighted its premotor...