Doug Kerr, MD, PhD

The chief medical officer at Dyne Therapeutics highlighted z-rostudirsen’s ability to restore dystrophin and improve function across DMD severity levels, setting the stage for a potential 2026 accelerated approval. [WATCH TIME: 4 minutes]

The chief medical officer at Dyne Therapeutics discussed the ACHIEVE trial’s early results, highlighting z-basivarsen’s potential to meaningfully improve muscle and CNS function in patients with myotonic dystrophy type 1. [WATCH TIME: 3 minutes]

The chief medical officer at Dyne Therapeutics discussed the scientific rationale and clinical progress of z-basivarsen, an antisense oligonucleotide therapy designed to correct mis-splicing in myotonic dystrophy type 1. [WATCH TIME: 5 minutes]

A panel of neurology experts detailed the importance that community leadership and creating a positive environment can do for patients with rare neurologic disorders [WATCH TIME: 5 minutes]

Sanford Siegel, Douglas Kerr, MD; and Benjamin Greenberg, MD, detailed the need for greater research on treatment strategies that repair myelin improve neurodegeneration, as well as expanded knowledge on how neuroimmune disorders present. [WATCH TIME: 5 minutes]

Benjamin Greenberg, MD; Douglas Kerr, MD; and Sanford Siegel provided perspectives on the gaps in the epidemiology and treatment paradigm for patients with rare neuroimmune disorders. [WATCH TIME: 3 minutes]

An expert neurology panel detailed some of the changes in therapeutic strategies and approaches to treating rare neuroimmune disorders, including the steps taken during the acute phase. [WATCH TIME: 6 minutes]

Several experts in neuroimmune disorders discuss the mechanisms of action that underlie these disorders, and how the perception of their pathology has changed. [WATCH TIME: 7 minutes]

A group of panelists discussed the history of the Siegel Rare Neuroimmune Association, and the progress made on rare neuroimmune disorders in the nearly 30 years since its existence.