A5036695794- Alkaline Phosphatase Research Studies
- Peripheral Neuropathies and Disorders
- Myasthenia Gravis and Thymoma
- Blood Pressure and Hypertension Studies
- Heterotopic Ossification and Related Conditions
- Pharmacology and Obesity Treatment
- Multiple Sclerosis Research Studies
- Kidney Stones and Urolithiasis Treatments
- Antifungal resistance and susceptibility
- Retinal Development and Disorders
- Biochemical and Molecular Research
- Heart Failure Treatment and Management
- Neurogenetic and Muscular Disorders Research
- Bone health and treatments
- Bone health and osteoporosis research
- Bone and Joint Diseases
- Vitamin D Research Studies
- Cardiovascular Syncope and Autonomic Disorders
- Glaucoma and retinal disorders
- Acute Kidney Injury Research
- Williams Syndrome Research
- thermodynamics and calorimetric analyses
- Diabetes Management and Research
- Sympathectomy and Hyperhidrosis Treatments
- Neonatal Health and Biochemistry
Alnylam Pharmaceuticals (United States)
2020-2021
Alexion Pharmaceuticals (United States)
2016-2020
University at Buffalo, State University of New York
2020
Stanford University
2020
Fondazione IRCCS Istituto Neurologico Carlo Besta
2020
Merck & Co., Inc., Rahway, NJ, USA (United States)
2010-2016
Clinical Research Institute
2011
Duke University Hospital
2011
Duke Medical Center
2011
University of Brescia
2011
Neuromyelitis optica spectrum disorder (NMOSD) is a relapsing, autoimmune, inflammatory that typically affects the optic nerves and spinal cord. At least two thirds of cases are associated with aquaporin-4 antibodies (AQP4-IgG) complement-mediated damage to central nervous system. In previous small, open-label study involving patients AQP4-IgG–positive disease, eculizumab, terminal complement inhibitor, was shown reduce frequency relapse.
Eculizumab is effective and well tolerated in patients with antiacetylcholine receptor antibody-positive refractory generalized myasthenia gravis (gMG; REGAIN; NCT01997229). We report an interim analysis of open-label extension REGAIN, evaluating eculizumab's long-term safety efficacy.Eculizumab (1,200 mg every 2 weeks for 22.7 months [median]) was administered to 117 patients.The profile eculizumab consistent no cases meningococcal infection were reported during the period. Myasthenia...
Background. Hypophosphatasia (HPP) is caused by loss-of-function mutation(s) of the gene that encodes tissue-nonspecific isoenzyme alkaline phosphatase (TNSALP). Consequently, cell-surface deficiency TNSALP phosphohydrolase activity leads to extracellular accumulation inorganic pyrophosphate, a natural substrate and inhibitor mineralization. Children with HPP can manifest rickets, skeletal pain, deformity, fracture, muscle weakness, premature deciduous tooth loss. Asfotase alfa recombinant,...
Primary hyperoxaluria type 1 (PH1) is a rare, progressive, genetic disease with limited treatment options. We report the efficacy and safety of lumasiran, an RNA interference therapeutic, in infants young children PH1.This single-arm, open-label, phase 3 study evaluated lumasiran patients aged <6 years PH1 estimated glomerular filtration rate >45 mL/min/1.73 m2, if ≥12 months, or normal serum creatinine, <12 months. The primary end point was percent change spot urinary oxalate to creatinine...
Cardiac troponin T (cTnT) elevation is common and a predictor of outcomes in patients with acute heart failure (AHF). The degree progression cTnT release during hospitalization AHF unclear. We evaluated the incidence relationship outcomes.The Placebo-controlled Randomized study selective A(1) adenosine receptor antagonist rolofylline for hospitalized volume Overload to assess Treatment Effect on Congestion renal funcTion (PROTECT) pilot was multicenter, double-blind AHF. Measurements were...
<h3>Objective</h3> To evaluate whether eculizumab helps patients with anti–acetylcholine receptor–positive (AChR+) refractory generalized myasthenia gravis (gMG) achieve the Myasthenia Gravis Foundation of America (MGFA) post-intervention status minimal manifestations (MM), we assessed patients9 throughout REGAIN (Safety and Efficacy Eculizumab in AChR+ Refractory Generalized Gravis) its open-label extension. <h3>Methods</h3> Patients who completed randomized controlled trial continued into...
Long-term data on enzyme replacement treatment of hypophosphatasia (HPP) are limited. To evaluate efficacy and safety asfotase alfa in patients aged ≤5 years with HPP followed for up to 6 years. Phase 2 open-label study (July 2010 September 2016). Twenty-two sites; 12 countries. Sixty-nine [median (range) age: 16.0 (0.02 72) months] severe sign/symptom onset before age months. Asfotase mg/kg three times/week or 1 six subcutaneously. Primary measure: Radiographic Global Impression Change...
Abstract Background The efficacy and tolerability of eculizumab were assessed in REGAIN, a 26-week, phase 3, randomized, double-blind, placebo-controlled study anti-acetylcholine receptor antibody-positive (AChR+) refractory generalized myasthenia gravis (gMG), its open-label extension. Methods Attainment ‘minimal symptom expression’ was evaluated using patient-reported outcome measures gMG symptoms [MG activities daily living scale (MG-ADL), 15-item MG quality life questionnaire (MG-QOL15)]...
Signaling of vision to the brain starts with retinal phototransduction cascade which converts visible light from environment into chemical changes. Vision impairment results when mutations inactivate proteins cascade. A severe monogenically inherited blindness, Leber congenital amaurosis (LCA), is caused by in GUCY2D gene, leading a molecular defect production cyclic GMP, second messenger phototransduction. We studied two patients GUCY2D-LCA who were undergoing gene augmentation therapy....
ABSTRACT Hypophosphatasia (HPP) is the heritable metabolic disease characterized by impaired skeletal mineralization due to low activity of tissue-nonspecific isoenzyme alkaline phosphatase. Although HPP during growth often manifests with distinctive radiographic features, no validated method was available quantify them, including changes over time. We created Radiographic Global Impression Change (RGI-C) scale assess in burden pediatric HPP. Site-specific pairs radiographs newborns,...
Background: In the phase III eculizumab for refractory generalized myasthenia gravis REGAIN study [ClinicalTrials.gov identifier: NCT01997229] and its open-label extension (OLE) NCT02301624], patients with treatment-refractory antiacetylcholine receptor antibody-positive had clinically meaningful improvements versus placebo. This subgroup analysis evaluated data from a recent history of chronic intravenous immunoglobulin (IVIg) use before entry. Methods: The comprised who received IVIg at...
The terminal complement inhibitor eculizumab was shown to improve myasthenia gravis-related symptoms in the 26-week, phase 3, randomized, double-blind, placebo-controlled REGAIN study (NCT01997229). In this 52-week sub-analysis of open-label extension (NCT02301624), eculizumab's efficacy and safety were assessed 11 Japanese 88 Caucasian patients with anti-acetylcholine receptor antibody-positive refractory generalized gravis. For who had received placebo during REGAIN, treatment resulted...
Objective: To evaluate the safety and efficacy of sitagliptin when added to treatment patients with type 2 diabetes mellitus (T2DM) inadequate glycemic control on acarbose monotherapy.Research design methods: This was a multicenter, randomized, placebo-controlled, double-blind clinical trial. Patients (N = 381) T2DM (glycated hemoglobin [HbA1c] ≥ 7.0% ≤10.0%) monotherapy (at least 50 mg three times daily) were randomized in 1:1 ratio receive addition 100 or matching placebo once daily for 24...
Japanese patients with uncontrolled essential hypertension received single-blind losartan 50 mg/hydrochlorothiazide 12.5 mg (L50/H12.5) for 8 weeks. Patients whose blood pressure (BP) remained were randomized double-blind to fixed-dose mg/amlodipine 5 (L50/H12.5/A5) or L50/H12.5 weeks followed by open-label L50/H12.5/A5 44 Adverse events assessed. After weeks, diastolic and systolic BP reduced significantly more versus (both p < 0.001). Mean changes in sustained was well-tolerated improved...
To modify the Performance-Oriented Mobility Assessment-Gait (POMA-G) subtest and validate this modified POMA-G (mPOMA-G) in children with hypophosphatasia (HPP), a rare metabolic disorder that can manifest musculoskeletal symptoms impair mobility ambulation.Based on feedback from an expert panel, was by removing gait initiation/path assessments expanding rating scale for step length/continuity to capture aspects of observational analysis relevant HPP. Three trained physical therapists used...