A5077749574- Amyotrophic Lateral Sclerosis Research
- Parkinson's Disease Mechanisms and Treatments
- Autism Spectrum Disorder Research
- Child Nutrition and Feeding Issues
- Hemoglobinopathies and Related Disorders
- Neurogenetic and Muscular Disorders Research
- Iron Metabolism and Disorders
- Family and Disability Support Research
- Balance, Gait, and Falls Prevention
- Health Systems, Economic Evaluations, Quality of Life
- Mindfulness and Compassion Interventions
- Stroke Rehabilitation and Recovery
- Folate and B Vitamins Research
- Neurofibromatosis and Schwannoma Cases
- Traumatic Brain Injury Research
- Dysphagia Assessment and Management
- Genetics and Neurodevelopmental Disorders
- Neurological disorders and treatments
- Cerebral Palsy and Movement Disorders
- Musculoskeletal pain and rehabilitation
- Pharmacological Effects and Toxicity Studies
- Statistical Methods in Clinical Trials
- Resilience and Mental Health
- Psychosomatic Disorders and Their Treatments
- Complementary and Alternative Medicine Studies
Massachusetts General Hospital
2016-2025
Harvard University
2016-2025
Clinical Research Institute
2013-2025
Boston University
2010-2024
Lemuel Shattuck Hospital
2010-2024
University of Rochester Medical Center
2023
Toronto Western Hospital
2023
University of Toronto
2023
Public Health Ontario
2023
University of California, San Diego
2023
Dispersal affects community dynamics and vegetation response to global change. Understanding these effects requires descriptions of dispersal at local regional scales statistical models that permit estimation. Classical describe or long-distance dispersal, but not both. The lack methods means have rarely been fitted seed in closed forests. We present a mixture model assumes range disperal patterns, both long distance. bivariate Student's t "2Dt" follows from an assumption the distance...
Recruitment limitation of tree population dynamics is poorly understood, because fecundity and dispersal are difficult to characterize in closed stands. We present an approach that estimates seed production under canopies four limitations on recruitment: density location, fecundity, dispersal, establishment. Consistent obtained for 14 canopy species using 5 yr census data from 100 traps several thousand mapped trees seedlings five southern Appalachian forest stands span gradients elevation...
Mutations in SOD1 cause 13% of familial amyotrophic lateral sclerosis. In the Gly93Ala rat model sclerosis, antisense oligonucleotide ISIS 333611 delivered to CSF decreased mRNA and protein concentrations spinal cord tissue prolonged survival. We aimed assess safety, tolerability, pharmacokinetics after intrathecal administration patients with SOD1-related sclerosis.In this randomised, placebo-controlled, phase 1 trial, we by infusion using an external pump over 11·5 h at increasing doses...
Sodium phenylbutyrate and taurursodiol have been found to reduce neuronal death in experimental models. The efficacy safety of a combination the two compounds persons with amyotrophic lateral sclerosis (ALS) are not known.
Autism spectrum disorder (ASD), characterized by both impaired communication and social interaction, stereotypic behavior, affects about 1 in 68, predominantly males. The medico-economic burdens of ASD are enormous, no recognized treatment targets the core features ASD. In a placebo-controlled, double-blind, randomized trial, young men (aged 13-27) with moderate to severe received phytochemical sulforaphane (n = 29)--derived from broccoli sprout extracts--or indistinguishable placebo 15)....
Aggregated α-synuclein plays an important role in Parkinson's disease pathogenesis. Cinpanemab, a human-derived monoclonal antibody that binds to α-synuclein, is being evaluated as disease-modifying treatment for disease.In 52-week, multicenter, double-blind, phase 2 trial, we randomly assigned, 2:1:2:2 ratio, participants with early receive intravenous infusions of placebo (control) or cinpanemab at dose 250 mg, 1250 3500 mg every 4 weeks, followed by active-treatment dose-blinded extension...
IMPORTANCE Convergent biological, epidemiological, and clinical data identified urate elevation as a candidate strategy for slowing disability progression in Parkinson disease (PD).OBJECTIVE To determine the safety, tolerability, urate-elevating capability of precursor inosine early PD to assess its suitability potential design features disease-modification trial. DESIGN, SETTING, AND PARTICIPANTSThe Safety Urate Elevation (SURE-PD) study, randomized, double-blind, placebo-controlled,...
Abstract An orally administered, fixed‐dose coformulation of sodium phenylbutyrate‐taurursodiol (PB‐TURSO) significantly slowed functional decline in a randomized, placebo‐controlled, phase 2 trial ALS (CENTAUR). Herein we report results long‐term survival analysis participants CENTAUR. In CENTAUR, adults with were randomized 2:1 to PB‐TURSO or placebo. Participants completing the 6‐month (24‐week) eligible receive open‐label extension. all‐cause mortality (35‐month maximum follow‐up...
Urate elevation, despite associations with crystallopathic, cardiovascular, and metabolic disorders, has been pursued as a potential disease-modifying strategy for Parkinson disease (PD) based on convergent biological, epidemiological, clinical data.To determine whether sustained urate-elevating treatment the urate precursor inosine slows early PD progression.Randomized, double-blind, placebo-controlled, phase 3 trial of oral in PD. A total 587 individuals consented, 298 not yet requiring...
Abstract Amyotrophic lateral sclerosis (ALS) involves progressive motor neuron loss, leading to paralysis and death typically within 3–5 years of diagnosis. Dysfunctional astrocytes may contribute disease glial cell line-derived neurotrophic factor (GDNF) can be protective. Here we show that human neural progenitor cells transduced with GDNF (CNS10-NPC-GDNF) differentiated protected spinal neurons were safe in animal models. CNS10-NPC-GDNF transplanted unilaterally into the lumbar cord 18...
Increasing atmospheric carbon dioxide is responsible for climate changes that are having widespread effects on biological systems. One of the clearest earlier onset spring and lengthening growing season. We designed present study to examine interactive timing dormancy release seeds with low high CO2 biomass, reproduction, phenology in ragweed plants (Ambrosia artemisiifolia L.), which produce highly allergenic pollen. released from at three 15-day intervals grew climate-controlled...
Adults with beta thalassemia major frequently have low BMD, fractures, and bone pain. The purpose of this study was to determine the prevalence pain in all syndromes childhood, adolescence, adulthood, associations BMD fractures pain, etiology disease thalassemia. Patients Thalassemia Clinical Research Network, > or =6 yr age, no preexisting medical condition affecting mass requiring steroids, participated. We measured spine femur whole body BMC by DXA assessed vertebral abnormalities...
The objective of this study was to characterize the diagnostic timelines and their predictors in people with amyotrophic lateral sclerosis (ALS). Patients were identified through ALS billing codes. Time from presenting symptom first doctor visit, visit suspected diagnosis, confirmed diagnosis (total time) collected. Regression models used analyze delay. Three hundred four patients included analysis. Median total time 11.5 months. Diagnostic longer age > 60 years (p < 0.001), sporadic =...
This study aimed to determine differences in the rates of growth, endocrine- and calcium-related abnormalities various thalassemia syndromes North America treated with current therapies. Medical history, physical examinations blood urine collections were obtained from patients all age 6 years older Thalassemia Clinical Research Network. 361 subjects, 49% male, mean 23.2 (range 6.1-75 years) studied. Approximately 25% children adults, regardless syndrome, had short stature. Overall growth was...
Abstract Purpose: Resistance to temozolomide chemotherapy is partly mediated by O6-methylguanine-DNA methlytransferase (MGMT). Protracted treatment with potentially overcomes MGMT resistance and improves outcome. We conducted a phase II study of protracted daily in adults low-grade gliomas. Experimental Design: Patients newly diagnosed oligodendroglioma or oligoastrocytoma MIB-1 index &gt;5% recurrent gliomas received (75 mg/m2/day 11-week cycles 7 weeks on/4 off). Treatment continued...
An analysis of a prospective cohort individuals with sickle cell anemia (SCA), enrolled from birth through adulthood, was conducted to determine if asthma is risk factor for death in SCA. All-cause mortality determined participants after adjusting known factors The study included 1,963 who were followed 18,495 patient-years. After controlling established factors, SCA and had more than two-fold higher (hazard ratio 2.36, 95% CI 1.21 4.62, p=0.01). To summarize,
More effective treatments are needed for negative symptoms of schizophrenia, which typically chronic, disabling, and costly. Negative have previously been associated with reduced blood folate levels, especially among patients low-functioning variants in genes that regulate metabolism, suggesting the potential utility supplementation.To determine whether folic acid plus vitamin B12 supplementation reduces schizophrenia functional folate-related influence treatment response.Parallel-group,...
Aliment Pharmacol Ther 2011; 34: 1328–1336 Summary Background Many patients with ulcerative colitis (UC) and Crohn’s disease (CD) complain of significant fatigue. To date, no instrument to measure fatigue has been validated in a US inflammatory bowel (IBD) population. Aim determine the reliability validity Functional Assessment Chronic Illness Therapy‐Fatigue (FACIT‐F) scale IBD. Methods A total 209 IBD completed 13 items FACIT‐F, alongside laboratory testing activity assessment. Internal...