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Small Molecules in Development for the Treatment of Spinal Muscular Atrophy.
Spinal muscular atrophy (SMA) is an autosomal recessive neurodegenerative disease resulting from pathologically low levels of survival motor neuron (SMN) protein. The majority of mRNA from the SMN2Expand
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Restored glial glutamate transporter EAAT2 function as a potential therapeutic approach for Alzheimer’s disease
Takahashi et al. demonstrate that restoring glial glutamate transporter EAAT2 function improves cognitive functions and synaptic integrity while reducing amyloid plaques in a sustained fashion afterExpand
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trans-1-[(2-Phenylcyclopropyl)methyl]-4-arylpiperazines: mixed dopamine D(2)/D(4) receptor antagonists as potential antipsychotic agents.
The dopaminergic receptor profile of a series of trans-1-[(2-phenylcyclopropyl)methyl]-4-arylpiperazines was examined. Aromatic substitution patterns were varied with the goal of identifying aExpand
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Primary epineural repair of the ulnar nerve in children.
Transectional nerve injuries are uncommon in children. We report the outcome of 19 children aged < or = 13 years with acute transectional injuries to the ulnar nerves who were treated by primaryExpand
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The Role of Fluorine in the Discovery and Optimization of CNS Agents: Modulation of Drug-Like Properties
Publisher Summary This chapter highlights the role of fluorine in the discovery and optimization of central nervous system (CNS) agents. It also describes the strategic placement of fluorine intoExpand
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Discovery of N-(1-ethylpropyl)-[3-methoxy-5-(2-methoxy-4-trifluoromethoxyphenyl)-6-methyl-pyrazin-2-yl]amine 59 (NGD 98-2): an orally active corticotropin releasing factor-1 (CRF-1) receptor
The design, synthesis, and structure-activity relationships of a novel series of pyrazines, acting as corticotropin releasing factor-1 (CRF-1) receptor antagonists, are described. SyntheticExpand
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Discovery of a Small Molecule Probe That Post-Translationally Stabilizes the Survival Motor Neuron Protein for the Treatment of Spinal Muscular Atrophy.
Spinal muscular atrophy (SMA) is the leading genetic cause of infant death. We previously developed a high-throughput assay that employs an SMN2-luciferase reporter allowing identification ofExpand
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A Stearoyl–Coenzyme A Desaturase Inhibitor Prevents Multiple Parkinson Disease Phenotypes in α‐Synuclein Mice
Parkinson disease (PD) has useful symptomatic treatments that do not slow the neurodegenerative process, and no significant disease‐modifying treatments are approved. A key therapeutic target in PDExpand
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Primary Epineural Repair of the Median Nerve in Children
We report 18 children (12 boys and six girls) with a mean age of 6.1 years who sustained lacerations of the median nerve. Associated injuries to other structures occurred in 15 children. TreatmentExpand
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From arylureas to biarylamides to aminoquinazolines: discovery of a novel, potent TRPV1 antagonist.
Bioisosteric replacement of piperazine with an aryl ring in lead VR1 antagonist 1 led to the biarylamide series. The development of B-ring SAR led to the conformationally constrained analog 70. TheExpand
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