Montreal Neurological Institute and Hospital
Clinical Research Unit
Here is Dr Genge’s short bio: Dr. Angela Genge has been the Executive Director of the Clinical Research Unit at the Montreal at the Montreal Neurological Institute since 2004. Since 2020, she has also served as Chief Medical Officer for QurAlis and Scientific Director of CATALIS. Her expertise and ground-breaking work in the introduction of innovative therapies in the rare disease space has led her to be a sought-after member of numerous advisory boards for companies developing therapies for these diseases. Clinically, she is the Director of the Neuro ALS clinical program and multidisciplinary clinic, and a neuromuscular neurologist. Throughout her career she has received numerous awards, most recently the 2018 Forbes Norris Award, the DIVA of Distinction Award, and the Governor General Diamond Jubilee Award.

Moderator of 1 Session

Session Type
SYMPOSIUM
Date
Wed, 16.03.2022
Session Time
04:15 PM - 06:15 PM
Room
ONSITE: 133-134

Presenter of 1 Presentation

C9ORF72 and TDP43 Related Motor Neuron Diseases: Current approaches in drug development

Session Type
SYMPOSIUM
Date
Wed, 16.03.2022
Session Time
04:15 PM - 06:15 PM
Room
ONSITE: 133-134
Lecture Time
05:45 PM - 06:00 PM

Abstract

Aims

Objectives- To describe current efforts in ALS drug development to correct the consequences of TDP43 pathology by tackling different underlying targets.

Methods

Methods- Review of current clinical development programs, including biomarker development that are focusing on pathways related to TDP43 pathology

Results

Results- Several programs are currently targeting TDP 43 mechanisms of disease in both ALS and FTD. The results of these current programs will provide valuable insights into disease mechanisms in addition to determining target engagement and ultimately efficacy of the compounds being studied in different programs. Additionally biomarker development and validation will allow more efficient trial design in the future

Conclusions

Conclusions TDP43 pathology is a viable target in ALS drug development. Lessons learned will be applicable to frontotemporal dementia and beyond.
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