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Biomedical Sciences Research Seminar Series

Targeting muscle and metabolic pathologies to develop new treatment strategies for spinal muscular atrophy

Atomic structure of a biological molecule
Seminars

As part of the School of Science and Technology Biomedical Sciences Research Centre Seminar Series, Melissa Bowerman, Keele University presents: Targeting muscle and metabolic pathologies to develop new treatment strategies for spinal muscular atrophy

  • From: Wednesday 6 November 2019, 1.10 pm
  • To: Wednesday 6 November 2019, 2 pm
  • Location: ERD 282, Erasmus Darwin, Nottingham Trent University, Clifton Campus, Clifton Lane, Nottingham, NG11 8NS
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Event details

As part of the School of Science and Technology Biomedical Sciences Research Centre Seminar Series, Melissa Bowerman, Keele University presents: Targeting muscle and metabolic pathologies to develop new treatment strategies for spinal muscular atrophy

Abstract

Spinal muscular atrophy (SMA) is a devastating neuromuscular disease caused by reduced levels of the survival motor neuron (SMN) protein and characterized by motoneuron and muscle loss. Patients with the severe form typically die before the age of two while children with milder forms live into adulthood with limited motor abilities. The Food and Drug Administration (FDA) and European Medicines Agency (EMA) have recently approved the first SMN replacement strategy and additional promising SMN-enhancing gene therapies and small molecules are currently being evaluated. However, emerging results from pre-clinical and clinical studies highlight the need for approaches that will also include SMN–independent and systemic strategies. We and others have demonstrated that SMA patients and animal models display various metabolic and muscle pathologies that contribute to disease manifestation and progression. Our overall aim is to identify novel SMN-independent therapeutic strategies that ameliorate metabolic and/or muscle perturbations and that can be used in combination with clinically relevant SMN gene therapies for the treatment of all SMA patients.

Hosted by Craig Doig

All welcome.

For any enquiries please contact Dr Amanda Coutts

Location details

Room/Building:

ERD 282, Erasmus Darwin

Address:

Nottingham Trent University
Clifton Campus
Clifton Lane
Nottingham
NG11 8NS

Past event

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