Spinal Muscular Atrophy: Novel Approaches for Treatment
Spinal muscular atrophy (SMA) is an autosomal recessive neurodegenerative disorder characterized by progressive muscle wasting and eventual loss of muscle function due to severe motor neuron dysfunction. It is one of the leading heritable causes of infant mortality worldwide and has a carrier frequency of approximately 1 in 40 people. Join Kevin Hodgetts of the Harvard Medical School as he discusses the pipeline of therapies for SMA which encompass four different strategies: (i) modulation of SMN2 encoded full-length protein levels; (ii) SMN1 gene replacement; (iii) neuroprotection; and (iv) muscle strength and function. During this presentation he will reveal the exciting recent approval of the antisense oligonucleotide, Spinraza, which is the first step to a cure for this disorder.
What You Will Learn
- What is Spinal Muscular Atrophy and what are the causes
- What is the current SMA Drug Discovery Pipeline
- The medicinal chemistry optimization of molecules that stabilize the survival motor neuron (SMN) protein and increases the SMN protein transcription
- "Small Molecules in Development for the Treatment of Spinal Muscular Atrophy" - Journal of Medicinal Chemistry Article
- "Discovery of a Small Molecule Probe That Post-Translationally Stabilizes the Survival Motor Neuron Protein for the Treatment of Spinal Muscular Atrophy" - Journal of Medicinal Chemistry Article
- "Identification of a Maleimide-Based Glycogen Synthase Kinase-3 (GSK-3) Inhibitor, BIP-135, That Prolongs the Median Survival Time of Δ7 SMA KO Mouse Model of Spinal Muscular Atrophy" - ACS Chemical Neuroscience Article
- "Discovery and Optimization of Small Molecule Splicing Modifiers of Survival Motor Neuron 2 as a Treatment for Spinal Muscular Atrophy" - Journal of Medicinal Chemistry Article
- "Specific Correction of Alternative Survival Motor Neuron 2 Splicing by Small Molecules: Discovery of a Potential Novel Medicine To Treat Spinal Muscular Atrophy" - Journal of Medicinal Chemistry Article
- "Synthesis and Biological Evaluation of Novel 2,4-Diaminoquinazoline Derivatives as SMN2 Promoter Activators for the Potential Treatment of Spinal Muscular Atrophy" - Journal of Medicinal Chemistry Article
- "Design of Potent mRNA Decapping Scavenger Enzyme (DcpS) Inhibitors with Improved Physicochemical Properties To Investigate the Mechanism of Therapeutic Benefit in Spinal Muscular Atrophy (SMA)" - Journal of Medicinal Chemistry Article
The Fine Print
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