Defeat Duchenne Family Forum | Calgary, Alberta | Saturday, November 2, 2019
Thank you to the families, sponsors, and speakers for supporting the inaugural Defeat Duchenne Family Forum in Western Canada presented by PTC Therapeutics. This family-oriented event took place on Saturday, November 2, 2019, in Calgary, Alberta and saw more than 20 families affected by Duchenne muscular dystrophy come together with world-renowned researchers, clinicians, and industry professionals for a day of education, inspiration, and hope.
Click here to take a look at the Defeat Duchenne Family Forum photo reel thanks to Brian Buchsdruecker of Bookstrucker Photography. Don’t forget to share and tag Jesse’s Journey on Twitter (@jesses_journey) and Facebook (/defeatduchenne) along with including the hashtag #defeatduchenne.
Did you miss the Defeat Duchenne Family Forum in Calgary, Alberta? Click here to watch the 11 speakers share insight and information about Duchenne research, clinical trials in Canada, standards of care, pharmaceutical progress, registries, advocacy. These videos and much more can be found on our YouTube channel.
We will be featuring leading Researchers in Duchenne from across North America;
Presentation: "Pharmacological Modulation of Muscle Stem Cells to Treat Duchenne Muscular Dystrophy"
Dr. Michael Rudnicki is a Senior Scientist and the Director of the Regenerative Medicine Program at the Ottawa Hospital Research Institute and is the Scientific Director of the Stem Cell Network.
He is an internationally recognized leader in molecular genetics and regenerative medicine whose research has transformed our understanding of muscle development and regeneration, and fueled the development of novel molecular and stem cell based approaches to the treatment of muscular dystrophy. Dr. Rudnicki’s key discovery was the identification and characterization of muscle stem cells. This work led to the study of adult regenerative myogenesis and set the stage for novel molecular and stem cell based approaches to the treatment of muscular dystrophy and other neuromuscular disorders.
Presentation: "Vision DMD: Vamorolone Drug Development for DMD"
Laura Hagerty, Ph.D, joined ReveraGen BioPharma in 2019 as a New Drug Application Coordinator. Prior to joining ReveraGen, she served as Scientific Portfolio Director at Muscular Dystrophy Association where she directed and managed strategic investment in research for Duchenne and related dystrophies. Hagerty spent nearly a decade working on preclinical drug development for Duchenne within Pharma
Presentation: "Patient Registries Improving Clinical Care in NMD and Update on CNDR"
Dr. Korngut, has led more than a dozen major clinical trials of new treatments for neuromuscular diseases. He also founded the Canadian Neuromuscular Disease Registry, which tracks patients across Canada, enabling clinical trials
Dr. Lawrence Korngut is an internationally recognized leader in the research and treatment of neuromuscular disease and is a passionate advocate for his patients
Presentation: "Exon Skipping and Gnome Editing: Emerging Therapies for DMD"
Currently, Dr. Yokota is a Professor at the Department of Medical Genetics, University of Alberta, holding the title of the Friends of Garrett Cumming Research & Muscular Dystrophy Canada HM Toupin Neurological Science Endowed Research Chair since 2011. He will talk about his latest research the on the “DNA Stitch process that could help up to 47% of the boys affected by DMD.
We are pleased to have from Alberta’s Children’s Hospital;
Presentation: "Emerging Therapies and Standards of Care"
Dr. Mah is a pediatric neurologist and the physician leader of the pediatric neuromuscular program at the Alberta Children's Hospital. She is currently involved in a number of clinical research studies in collaboration with other researchers both locally and internationally, that include clinical trials for pediatric neuromuscular and acquired demyelinating disorders.
Her research also includes clinical endpoints and biomarkers exploration, for pediatric neurologic and neuromuscular disorders related to pediatric neuromuscular diseases.
Presentation: "Physiotherapy: The Importance of Stretching"
Angela has been a physiotherapist for 32 years and she is the coordinator of the Brachial Plexus Program. She will talk about why PT is so important for DMD, talk about the different assessments they take in clinic and meaning behind them. Tips and tricks for parents and individuals with DMD on stretching at home.
Presentation: "Measured by Moments"
Brett Billmeyer is a member of the patient engagement team at PTC Therapeutics. He unknowingly began his career as a patient advocate when he was diagnosed with a rare genetic disorder in 2009. After his diagnosis, he found that there were very few resources and no approved treatment for this disorder. He knew that if he wanted to change the outcome of the disease, he had to advocate for himself and others with this disorder. In 2011, he found a clinical trial for his rare disease and was the first patient to participate in this trial. He participated in this trial for over four years until the drug was approved. He co-founded a patient advocacy organization to serve as a connection point between the patient community and the pharmaceutical company. He was the voice of his community. He traveled throughout the United States, Canada and Europe educating the medical community about the disease, which is often times misdiagnosed or undiagnosed. Over the past ten years, he has worked with several other rare disease communities and has helped them establish their own patient advocacy organizations. He has been working with the Duchenne muscular dystrophy community for the past four years. He continues to fight for the patient’s right of access to quality care and for their right to be treated with compassion and dignity.
Presentation: "Update on Sarepta Pipeline: Duchenne Muscular Dystrophy"
Jennifer LaCorte, MSN, Medical Science Liaison, received her nursing degree from the University of Maryland. She has been in healthcare for 25 years and has developed a strong background in neurology, neurotrauma, cardiology, and all aspects of research, both pre-clinical and clinical. After working for several years with heart failure patients, she was drawn to the Duchenne muscular dystrophy population. Jennifer quickly discovered a deep passion for those with Duchenne, the families, and caregivers.
Jennifer was dismayed to discover there were no definitive treatments for this progressive, debilitating disease, and knew there had to be a way to do more. She joined the Sarepta Therapeutics team as a Medical Science Liaison to further the development of genetic technologies that will improve and save the lives of those with Duchenne. While completely dedicated to the scientific development behind Duchenne muscular dystrophy, Jennifer’s true love is interacting with those with Duchenne and the families. Their courage and determination inspire her to do more, be more, and love more.
Presentation: "Clinical Research 101 & Updates on Givinostat for Italfarmaco"
Lauren Morgenroth has authored over a dozen research publications. She manages the Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study and has lead the working group on Duchenne genotypes based on disease-causing mutations in Duchenne muscular dystrophy.
Lauren has explored barriers to engagement in rare disease research and has chaired numerous committees for neuromuscular groups. She is currently on the National Society of Genetic Counselors and the Parent Project Muscular Dystrophy Scientific Advisory Committee.
Special presentation on Advocacy – why it is important and a “How to” HUB;
Presentation: "Introduction to Effective Advocacy: Learn How to Be Heard"
Ryan Clarke is the president of Advocacy Solutions. He helps engage and influence key decision makers through the development and implementation of impactful advocacy strategies. Ryan has spoken internationally, teaching and training thousands of people how to make their voices heard.
AND Joanne’s expertise is deeply rooted in healthcare and enhanced by roles directing communications for two national patient advocacy organizations. Joanne works at the federal and provincial levels to support drug approval and reimbursement efforts to ensure novel therapies are accessible to all Canadians.
Venue: Acclaim Hotel Calgary Airport