Clinical Trials and Drug Approval

Ask-the-Expert: A Community Portrait through Surveys and Insurance Claims Data

Presented on June 17th.

Do you have questions for DM experts? Join Sarah Howe, MBA, of the Marigold Foundation for an “Ask-the-Expert” discussion, A Community Portrait through Surveys and Insurance Claims Data! 

The patient voice is critical to understanding myotonic dystrophy (DM). As experts in their own right, those living with DM can inform research, drug development, and government decision-making. Join Sarah Howe, MBA, Program Manager of the Marigold Foundation, as she presents findings from the Christopher Project and a DM insurance claims data analysis project. These studies shed new light on the burden of disease and illustrate the importance of patient-centered research in the development of scientific, clinical, and policy solutions to help improve the lives of individuals and families living with DM.

Speakers to include:

Sarah Howe, MBA has worked in myotonic dystrophy for 15 years as the Program Manager for the Marigold Foundation. Marigold is a private Canadian foundation focused on therapeutic development, research, and strategic initiatives in myotonic dystrophy. Most recently, Sarah was Project Coordinator for the Christopher Project, a large survey of patients, families, and caregivers living with DM1 and DM2 aimed at understanding their unmet needs and elevating the patient voice in myotonic dystrophy. Click here to read the open access publication.

Click here to find all our upcoming “Ask-the-Expert” question sessions!

Arthex Biotech - Meet the DM Drug Developers

Presented on June 3rd, 2022.

Each month biotechnology, pharmaceutical and academic partners, large and small, working on treatments and a cure for myotonic dystrophy will sit down with our community to share their progress and answer your questions. Click here to learn more about Arthex Biotech.

Dr. Beatriz Llamusi Troisi and Dr. Judy Walker present on Arthex Biotech's lead investigational compound for myotonic dystrophy, ATX-01.

Click here to find all our upcoming Meet the DM Drug Developers dates.

Dyne Therapeutics - Meet the DM Drug Developers

Presented on April 1st, 2022.

Each month biotechnology, pharmaceutical and academic partners, large and small, working on treatments and a cure for myotonic dystrophy will sit down with our community to share their progress and answer your questions. Click here to learn more about Dyne Therapeutics.

Dr. Ashish Dugar, SVP and Global Head of Medical Affairs at Dyne Therapeutics, will present an overview of Dyne’s DM1 candidate, DYNE-101, including preclinical data and plans for the upcoming clinical trial. Q&A hosted by Molly White, Vice President, Medical Communications & Advocacy.

Click here to find all our upcoming Meet the DM Drug Developers dates!

 

AMO Pharma - Meet the DM Drug Developers

 

Presented on March 5th, 2021.

In August 2020, AMO Pharma published the results of a Phase 2 study utilizing AMO-02 (Tideglusib) for Childhood Onset DM1 in Pediatric Neurology. AMO Pharma is excited to announce they will be moving forward with the REACH-CDM Clinical Trial for AMO-02 after the US FDA and Health Canada approved their recently revised study protocols. Click here to learn more about AMO Pharma.

Each month biotechnology, pharmaceutical and academic partners, large and small, working on treatments and a cure for myotonic dystrophy will sit down with our community to share their progress and answer your questions. Click here to find all our upcoming Meet the DM Drug Developers dates.

About the Presenters

Joe Horrigan, Chief Medical Officer

Joe received his Sc.B. degree from Brown University and his medical degree from the University of Rochester. Joe has been a longstanding scientific advisor to FRAXA, he is a Board member of Rettsyndrome.org, and he is a member of the Angelman Syndrome Foundation Scientific Advisory Committee. Joe is also a Consulting Associate Professor at the Duke University Center for Autism and Brain Development in Durham, North Carolina. At Duke, he evaluates and treats patients in Duke’s tertiary care autism clinic. 

Joe is a pediatric neuropsychiatrist, and he has specialized in the treatment of children with complex neurodevelopmental disorders for more than 30 years. Joewas a Senior Director in the Neurosciences Medicines Development Center at GlaxoSmithKline, where he also co-founded and led the company-wide Medicines for Children Advisory Network that collaborated with all therapeutic areas in the company. Dr. Horrigan also served as Assistant Vice President and Head of Medical Research for Autism Speaks, the largest science and advocacy organization in the U.S. devoted to autism spectrum disorders (ASD). He subsequently served as Vice President of Clinical Development and Medical Affairs for Neuren Pharmaceuticals Limited, leading that company’s pioneering clinical development efforts in neurodevelopmental disorders such as Rett syndrome and Fragile X syndrome, as well as in traumatic brain injury. 

Mike Snape, Chief Scientific Officer

Dr Mike Snape received his BA with Honors at Oxford University, England and his PhD from London University after study at the Maudsley and Bethlem Hospital.  Mike presently holds an adjunct Professorship at CASE Western university.  He has more than 30 years of experience in pharmaceutical research and development and has managed relevant projects from pre-clinical through clinical stages of development.   He previously worked in CNS R&D at AstraZeneca and also has extensive experience of research and development projects in the field of CNS disorders in a small life scie4nce company environment having been Principal Scientist at Cerebrus Ltd and Associate Director at Vernalis, and was a founder of Neuropharm.  Mike has been an advisor to venture capital groups in the UK, the Welcome Trust and sits on the advisory council of multiple research foundations.

Mike initiated one of the first industry sponsored clinical projects in autism in 1997, lead the first industry sponsored multi-center studies of a core symptom of autism, and some of the first industry sponsored studies in Fragile X Syndrome.  More recently Mike was one of the founders of AMO Pharma Ltd.  AMO Pharma submitted the first successful application for Fast Track Status for congenital myotonic dystrophy program to be granted by the FDA. 

Emily Fantelli, Associate Director, Clinical Development

Emily is studied biology at Pacific Lutheran University and then did graduate studies at the University of Washington, Seattle.  After working as an Emergency Medical Technician Emily gained experience as a Clinical Research Co-ordinator at Charles River Clinical Services. 

Emily then moved on to work at The Geneva Foundation.  At the latter institution Emily gained extensive experience implementing technology-based interventions for the treatment of Combat PTSD (e.g. virtual reality environments, app based support) at Joint Base Lewis-McChord and Fort Bragg installations.  Emily also has experience in designing pharmacological interventions for neurological disorders (Traumatic Brain Injury) as well as working on developmental disorders.

 

Industry Updates and Q&A - 2019 Myotonic Annual Conference

Representatives from biotech and pharma companies will provide updates on their drug development efforts in the DM field, including companies new to the Myotonic Annual Conference. The industry updates are followed by the Q&A session where questions submitted throughout the conference are answered by a panel of DM Professionals.

Matt Disney, PhD., Expansion Therapeutics - Slides
Art Levin, PhD, Avidity Biosciences - Slides coming soon!
Laury Mignon, PhD, Ionis Therapeutics - Slides coming soon!
Mo Qatanani, PhD, Dyne Therapeutics - Slides

Living with DM: Patients Report on Changes Over Time (2018 MDF Annual Conference)

This interactive panel session was presented in a similar format to the Patient-Focused Drug Development meeting presented by MDF as a part of the 2016 MDF Annual Conference, and the CNS Endpoint Development discussion presented at the 2017 MDF Annual Conference. Panelists discussed the disease symptom changes they have experienced living with myotonic dystrophy, followed by facilitated audience participation. This session is intended to amplify and deepen our understanding of disease changes and progression from the perspective of individuals and families living with DM.

Moderated by Charles Thornton, MD, University of Rochester

PowerPoint Presentation

Click here for more presentations and videos from the 2018 MDF Annual Conference in Nashville, TN.