Drug Repurposing for Neuromuscular Diseases

SMA is a neurodegenerative disease characterised by the loss of lower motor neurons and is an incurable disease. SMA is the leading genetic cause of early childhood lethality, with an incidence of 1 in 6,000 to 10,000 live births and a carrier frequency of 1 in 35-40. Current therapeutic strategies under development are almost exclusively based on increasing functional SMN protein. Given the divergence in prognoses due to the complexity of the different SMA type designation, the application of a combination of non-SMN dependent and SMN restoring therapies, would likely be essential to treat SMA. Therefore, there is an urgent need to develop and assess new non-SMN therapeutics for this incurable disease, which could be particularly effective at early stages of SMA. Modelis’ mission is to accelerate drug discovery for human genetic diseases and they are investing a large portion of their activities to drug repurposing. The current project will allow fastening the identification of already approved drugs for SMA using simple fish models of the disease in order to translate them quickly to the clinic

Faculty Supervisor:

Alex Parker;Pierre Drapeau

Student:

Estefania Carrillo

Partner:

Modelis inc.

Discipline:

Medicine

Sector:

Life sciences

University:

Université de Montréal