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Understanding Clinical Trials and Myotonic Dystrophy

Veröffentlicht: Oktober 21, 2026

Originally presented on October 13, 2026.

This presentation and question and answer session is designed to help our community better understand the clinical trial process! Learn more about what recent clinical trial results mean for future treatments and therapies for myotonic dystrophy (DM).

We want to hear from you! Take our quick 5-minute survey to share your questions, thoughts, and feelings about recent DM clinical trial news. Your feedback will help MDF understand what questions the community has and what information would be most helpful as clinical trials continue to develop. Take the survey on clinical trials! >>>

About the Presenters:

Daniel Grant, PhD, Novartis
Daniel Grant, PhD, is Global Program Head at Novartis, where he leads global development of del-desiran for myotonic dystrophy type 1 (DM1). He brings 15 years of drug development experience and has helped take new neuromuscular medicines from clinical development through regulatory approval. In his current role, he leads cross-functional strategy across clinical, regulatory, and medical teams, with a focus on bringing meaningful therapies to people living with neuromuscular disease.

Johanna Hamel, MD
Dr. Hamel is an Associate Professor of Neurology, Pathology and Laboratory Medicine and specializes in the diagnosis and treatment of neuromuscular diseases. Dr. Hamel cares for patients with acquired and hereditary neuromuscular diseases in clinic, with special expertise in myotonic dystrophy (DM) type 1 and type 2 and FSHD. She also performs electrodiagnostic studies in the EMG lab and teaches residents about neuromuscular diseases. She graduated from medical school at the Martin-Luther-University Halle-Wittenberg in Germany and worked as a neurology resident and researcher at the Charité in Berlin before completing a neurology residency and a clinical neuromuscular fellowship at the University of Rochester.

John W. Day, MD, PhD
Dr. Day relocated to Stanford University, as Professor of Neurology, Pediatrics and Pathology, in 2011 in order to build a comprehensive center for understanding and treating muscular dystrophy, serving as Director of Stanford’s Neuromuscular Medicine Program in the Department of Neurology and Neurological Sciences. Dr. Day remains an active member of the University of Minnesota collaborations he helped forge as Director of Minnesota’s Paul and Sheila Wellstone Muscular Dystrophy Center. He is working to integrate California and Minnesota resources with the international network of myotonic dystrophy research to assure that this most common form of muscular dystrophy is conquered as soon as possible.

Andy Rohrwasser
Dr. Andy Rohrwasser joined MDF in November 2023. He brings with him a broad and diverse background in academic science, venture backed start-ups and public health. Andy’s domain expertise and passion are rare diseases, genetics and genomics, and newborn screening. Andy implemented gene agnostic, next-generation sequencing as a universal second tier newborn screening modality. Under his leadership, Utah was the first state in the US to implement population wide screening for Spinal Muscular Atrophy (SMA).

MDF Resources referenced in this video:

Questions or comments? Contact MDF at 415-800-7777 or at info@myotonic.org.