An MDF community member and mother describes the medical team she has assembled for her daughter, Kate.

Originally presented on April 15th, 2022.

Do you have questions about brain-fog and the cognitive issues associated with DM1? Join DM expert Benjamin Gallais, PhD, of the the Center for the Study of Living Conditions and the Needs of the Population (ECOBES) for an “Ask-the-Expert” session on Key Neuropsychological Features of DM1.

View the open access article, Instrumental activities of daily living in adults with the DM1 childhood phenotype: going beyond motor impairments, referenced during the presentation.

Doctor Benjamin Gallais obtained his doctorate in clinical psychology from the University of Paris 8 in 2010 and received an MDF Research Fellowship in 2016. A clinical psychologist in France, his country of origin, he then completed a postdoctoral internship with the Interdisciplinary Research Group on Neuromuscular Diseases (GRIMN ) in the Saguenay, in order to deepen his knowledge of myotonic dystrophy type 1. Today, his expertise in this neuromuscular disease is recognized internationally. Specialized in health psychology and neuropsychology, Dr. Gallais is interested in the characterization of personality and damage to cognitive functions in neuromuscular diseases, their evolution and their impact on autonomy and social participation. In addition, his work focuses on the consequences of damage to the central nervous system, such as fatigue and motivational disorders. Dr. Gallais joined the ÉCOBES team in 2018.

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

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.

In 2017, MDF finally achieved one of its goals for the community when congenital myotonic dystrophy was added to the Compassionate Allowance Program for Social Security Disability. Join us for an informational webinar with SSA representative Deborah Dennis, who will explain how this new development affects the DM community and what will change in the application process going forward. Click here to download the Power Point Presentation.

Community members discuss daily living strategies for motivating their adult children living with juvenile-onset DM1.

Please note: This is an audio recording only. To view the accompanying presentation, please click here.

Community members Penni Warford, Sarah Clarke and Ann Spaulding discuss best practices for planning and implementing your child's Individualized Education Plan.

This is an audio recording only. To see the handout that accompanied this presentation, please click here.

Dr. Craig Campbell, MD, of Western University in Ontario, Canada, has a discussion with audience members about congenital DM. In the video, Dr. Campbell does not follow a set slide show, but he created slides for additional viewing information. Please click here to see his slides.

Community-led session led by Sarah Berman, Erica Kelly, and Catherine Wycoff, DPT, GCFP, ABMCP. Parents of children living with DM and a hippotherapy specialist discuss the benefit that this type of therapy can often have.

View related web resources from this presentation

This webinar is presented by a group of moms offering their knowledge and experience raising children with congenital DM. This webinar includes informed, practical suggestions from alleviating gastro-intestinal challenges with proper diet and natural supplements to advocating for your child's Individualized Education Program (IEP).