Active Research Studies
Funding to support the operation and expansion of the CMD-TR
Institution: Medical College of Wisconsin
Investigator: Michael Lawlor, MD
Diaphragm Contractility in Patients with Nemaline Myopathy
Study diaphragm function, assess exercise effect on diaphragm function, and assess effects of tirasemtiv.
Institution: VU University Medical Center
Investigator: Coen Ottenheijm, PhD, Baziel van Engelen and Jonne Doorduin
A Chemical-Biology Approach to Myofibril Stress Signaling: Zebrafish Component
Testing the most promising candidates in two zebrafish models of NM.
Institution: Children’s Hospital Boston & Harvard Medical School
Investigator: Alan Beggs, PhD
Pre-Clinical Evaluation of the FDA Drug Collection
Test FDA approved drugs on zebrafish then test promising ones on mice
Institution: Monash University
Investigator: Robert Bryson-Richardson,Tamar Sztal, Nigel Laing and Kristen Nowak
CRISPR/Cas9 Gene Editing: A Novel Therapeutic Strategy for Nemaline Myopathy
Reintroduce exon 55 in cell culture and then mouse models
Institution: Hospital for Sick Children
Investigator: James Dowling, MD, PhD and Hernan Gonorazky, MD
A Chemical-Biology Approach to Myofibril Stress Signaling
Work with Beggs (zebrafish) and Sweeney (mouse) to test small molecule that attenuates muscle stress in diaphragm.
Institution: Medical Faculty Mannheim
Investigator: Siegfried Labeit, MD, PhD, Alan Beggs, PhD and Lee Sweeney, PhD
Modulating Myosin to Improve Muscle Strength in Nebulin-Based Nemaline Myopathy
Increase myosin force generation by gene therapy and myosin activator that targets slow myosin.
Institution: University of Arizona
Investigator: Johan Lindqvist, PhD
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