Spinal Muscular Atrophy: Novel Approaches for Treatment

September 28, 2017 @ 2-3pm ET
Session 8 of the 2017 Drug Design and Delivery Symposium

Molecules in a cross-hair with a #7

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

The Fine Print

ACS Webinars® does not endorse any products or services. The views expressed in this presentation are those of the presenters and do not necessarily reflect the views or policies of the American Chemical Society.

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Kevin Hodgetts
Harvard Medical School

Alyson Weidmann
ACS Publications

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