Staff Scientist, Sanjana Lab, New York Genome Center, and NYU
Complement Inhibition in Myasthenia Gravis
Recent advancements have the potential to significantly improve the lives of individuals with myasthenia gravis and contribute to the broader understanding of autoimmune diseases
Therapeutic Potential of IGF-1 and MECP2 in Rett Syndrome
Research that is already underway will be necessary to confirm the safety and efficacy of this class of Rett syndrome therapies, and targeting IGF-1 may be a possibility for treating additional neurological disorders beyond Rett.
Activation of NRF2 and FXN in Friedreich Ataxia
Disease pathogenesis is attributed to oxidative stress—which can be regulated by NRF2, which, in turn, binds to antioxidant responsive elements in the promoter of the target gene FXN to control its expression.
Aβ Protofibril Elimination in Alzheimer Disease
One model of AD suggests that Aβ pathophysiology triggers downstream molecular pathways, including tauopathy, which lead to cortical neurodegeneration, and cognitive decline is further attributed to the associated neurocortical Aβ plaques.
Use of Nanoparticles to Combat Neurodegenerative Disease
Available neurodegenerative disease treatments are generally unsafe and ineffective at penetrating the blood-brain barrier, though the use of nanoparticles can provide improved penetration and exert a neuroprotective effect.
Potential Therapeutic Role of the Endocannabinoid System for Migraine
Identified implications of the endocannabinoid system in migraine physiology suggest that this pathway might hold therapeutic potential for some headache disorders.
Endoplasmic Reticulum and/or Mitochondrial-Dependent Neuronal Degeneration in ALS
Given the importance of mitochondrial and endoplasmic reticulum function in ALS, the disruption of intracellular mitochondria-endoplasmic reticulum contacts presents yet another avenue for neuronal degeneration—perhaps the primary point of underlying dysfunction.
PDE10A Inhibition in Tourette Syndrome
Despite progress in the understanding of Tourette syndrome and similar disorders, no clear cause of TS has been identified, nor are there treatment options that completely eliminate symptoms.
Stimulation Approaches to Epilepsy Treatment
With one-third of the 3.4 million US patients with epilepsy currently experience drug-resistant epilepsy, creating an urgent need for novel intervention strategies, such as stimulation approaches.
Serotonin Receptor Agonism in Dravet Syndrome
As Dravet syndrome is often nonresponsive to existing therapeutics, it can be a challenge to treat, and this refractory nature creates a great need for novel, innovative, and safe antiepileptic drugs for its treatment.
S1P Receptor Modulation in Multiple Sclerosis
Continued improvement in the pharmacodynamics and pharmacokinetics of S1PR modulators, particularly in modulator-receptor selectivity, should improve therapeutic efficacy while reducing the potential for AEs.
GHB as a GABA Receptor Agonist for Narcolepsy Therapy
γ-Hydroxybutyrate, known as GHB or oxybate, is a physiological compound present in the human body as both a precursor and degradation product of GABA.
Myostatin Inhibition in Amyotrophic Lateral Sclerosis
ALS has only recently been associated with causative or disease-modifying mutations in 20 genes that encode proteins with diverse functions, with promising therapeutic targets including proteins in pathways that regulate protein homeostasis.
Interleukin-6 Receptors in the Treatment of Neuromyelitis Optica Spectrum Disorder
NMOSD is associated with elevated levels of the pleiotropic cytokine interleukin-6, making the cytokine an apt therapeutic target.
PACAP Pathway and Its Role in Migraine
The PACAP pathway has shown promise as a potential novel therapeutic target in the treatment of migraine.
RGMa Inhibition for Repair and Protection in Multiple Sclerosis
Patients with MS have an urgent need for therapies that can reverse neurologic disability by promoting remyelination, and RGMa inhibition may help meet that need.
Inhibiting Cholesterol 24-Hydroxylase for Epilepsy Treatment
Despite the continual epilepsy treatment landscape growth, there are only a few if any approved therapies for rare developmental and epileptic encephalopathies.
Selective Inverse Agonists of the Histamine 3 Receptor as Treatment for Narcolepsy
Managing narcolepsy via behavioral strategies to improve excessive daytime sleepiness may be challenging but has shown some added efficacy when combined with pharmacological treatment for EDS.
Targeting Bruton Tyrosine Kinase for Multiple Sclerosis Treatment
An in-depth look into the expanding landscape of multiple sclerosis treatments that target bruton tyrosine kinase.
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