Search Results - "Church, Kathleen"
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Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy
Published in JAMA neurology (01-07-2021)“…This ongoing study assesses long-term safety and durability of response in infants with spinal muscular atrophy (SMA) type 1 after dosing with onasemnogene…”
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Territoriality modifies the effects of habitat complexity on animal behavior: a meta-analysis
Published in Behavioral ecology (18-04-2022)“…Abstract Augmenting habitat complexity by adding structure has been used to increase the population density of some territorial species in the wild and to…”
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Does increasing habitat complexity favour particular personality types of juvenile Atlantic salmon, Salmo salar?
Published in Animal behaviour (01-01-2018)“…The costs and benefits of a particular behavioural trait, such as boldness or aggression, may vary depending on the physical environment. We tested whether the…”
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Ideal despotic distributions in convict cichlids (Amatitlania nigrofasciata)? Effects of predation risk and personality on habitat preference
Published in Behavioural processes (01-01-2019)“…•Dominant fish preferred structurally complex versus open habitats.•Subordinate fish, but not dominants, altered habitat use in response to predation…”
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Effects of habitat complexity, dominance and personality on habitat selection: Ideal despotic cichlids
Published in Ethology (01-11-2019)“…Habitat structure can impede visibility and movement, resulting in lower resource monopolization and aggression. Consequently, dominant individuals may prefer…”
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Follistatin Gene Therapy for Sporadic Inclusion Body Myositis Improves Functional Outcomes
Published in Molecular therapy (05-04-2017)“…Sporadic inclusion body myositis, a variant of inflammatory myopathy, has features distinct from polymyositis/dermatomyositis. The disease affects men more…”
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AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort
Published in Journal of neuromuscular diseases (2019)“…Spinal muscular atrophy type 1 (SMA1) is the leading genetic cause of infant mortality for which therapies, including AVXS-101 (onasemnogene abeparvovec,…”
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Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
Published in The New England journal of medicine (02-11-2017)Get full text
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Evaluating potential effects of solar power facilities on wildlife from an animal behavior perspective
Published in Conservation science and practice (01-02-2021)“…Solar power is a renewable energy source with great potential to help meet increasing global energy demands and reduce our reliance on fossil fuels. However,…”
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A Novel Method of Genomic DNA Extraction for Cactaceae
Published in Applications in plant sciences (01-03-2013)“…Premise of the study: Genetic studies of Cactaceae can at times be impeded by difficult sampling logistics and/or high mucilage content in tissues. Simplifying…”
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Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled Trial
Published in JAMA neurology (01-09-2020)“…Micro-dystrophin gene transfer shows promise for treating patients with Duchenne muscular dystrophy (DMD) using recombinant adeno-associated virus serotype…”
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Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
Published in The New England journal of medicine (02-11-2017)“…Fifteen children with spinal muscular atrophy type 1 received gene-replacement therapy with a single dose of adeno-associated virus containing SMN. In marked…”
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Impact of Age and Motor Function in a Phase 1/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement Therapy
Published in Pediatric neurology (01-09-2019)“…This study characterizes motor function responses after early dosing of AVXS-101 (onasemnogene abeparvovec) in gene replacement therapy in infants with severe…”
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Health outcomes in spinal muscular atrophy type 1 following AVXS‐101 gene replacement therapy
Published in Pediatric pulmonology (01-02-2019)“…Background Spinal Muscular Atrophy type 1 (SMA1) is a rare genetic neuromuscular disease where 75% of SMA1 patients die/require permanent‐ventilation by 13.6…”
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Aggressive interactions between two invasive species: the round goby (Neogobius melanostomus) and the spinycheek crayfish (Orconectes limosus)
Published in Biological invasions (2017)“…The invasion success of introduced species may be limited by competitive interactions with phylogenetically unrelated invaders. The round goby ( Neogobius…”
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260 Gene transfer with rAAVrh74.MHCK7.SGCB increased β-sarcoglycan expression in LGMD type 2E patients
Published in Journal of neurology, neurosurgery and psychiatry (01-06-2022)“…These are initial findings of ongoing, phase 1 multiple ascending-dose gene transfer trial of ≤9 LGMD2E patients who received rAAVrh74.MHCK7.SGCB…”
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130 Safety, β-sarcoglycan expression and functional outcomes from systemic gene transfer of rAAVrh74.MHCK7.SGCB in LGMD2E/R4
Published in Journal of neurology, neurosurgery and psychiatry (01-06-2022)“…Limb-girdle muscular dystrophy type 2E/R4 (LGMD2E/R4) is caused by β-sarcoglycan (SGCB) gene mutations, resulting in loss of functional protein and…”
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Long‐term safety and functional outcomes of delandistrogene moxeparvovec gene therapy in patients with Duchenne muscular dystrophy: A phase 1/2a nonrandomized trial
Published in Muscle & nerve (01-01-2024)“…Introduction/Aims Delandistrogene moxeparvovec is indicated in the United States for the treatment of ambulatory pediatric patients aged 4 through 5 years with…”
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Long-term Safety and Efficacy in Patients with DMD 4 Years Post-Treatment with Delandistrogene Moxeparvovec (SRP-9001) in a Phase 1/2a Study (P3-8.006)
Published in Neurology (25-04-2023)“…Abstract only…”
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