Latest Conference Coverage

The pediatric epileptologist at Cleveland Clinic discussed the impact of ganaxolone’s approval for CDKL5 deficiency disorder and how it may create an opportunity for future pipeline agents. [WATCH TIME: 3 minutes]

Interim data from the phase 2 RESCUE-ALS trial open-label extension suggest that Clene Nanomedicine’s investigational agent, CNM-AU8, offered benefits to patients with ALS in terms of survival, in addition to prior results suggestive of slowed progression.

The Bushell Chair of Neurology at the University of Sydney shared his insight into the latest data on CNM-Au8 as a potential treatment for amyotrophic lateral sclerosis and the frameshift in treatment that it represents. [WATCH TIME: 3 minutes]

This year’s meeting, themed “the Great Neuro-Reunion,” will feature a dual virtual and in-person format, spanning 9 days and including more than 200 sessions and 11 plenaries, as well as featuring 2400 abstracts presentations.

The senior investigator in the Neuromuscular and Neurogenetic Disorders of Childhood Section at NINDS shared his perspective on the data coming out of neuromuscular research and the promise of genetic medicine. [WATCH TIME: 2 minutes]

The director of neuromuscular clinical trials at Columbia University Irving Medical Center shared her perspective on the discussions that took place at MDA 2022 and the importance of collaboration among clinicians and researchers.

The president and CEO of the Muscular Dystrophy Association spoke about the need for increased participation in neuromuscular disorder clinical trials and the rapid therapeutic progress the field is seeing. [WATCH TIME: 4 minutes]

Eleven of the 12 individuals demonstrated at least a 50% reduction in creatine kinase, with 75% of participants reaching twice the normal range, suggesting a reduction in muscle breakdown.

More than 90% of the 65-patient cohort had evidence of normal swallowing and had no aspiration or pneumonia aspiration events reported in the phase 1 START and phase 3 STRIVE-EU and STRIVE-US trials.

Kaplan-Meier estimates showed a 61% decreased risk of death on CNM-Au8 compared with predicted median survival from a published European Network to Cure ALS model.

The gene therapy from Novartis showed that it was both efficacious and well-tolerated in presymptomatic patients with SMA with 3 copies of SMN2, with no patients experiencing treatment-related serious adverse events.

Neurology News Network for the week ending March 19, 2022.

The chairman of the department of neurology at George Washington University discussed the reasons to be optimistic, but cautiously, about the expanding myasthenia gravis treatment toolbox. [WATCH TIME: 3 minutes]

After a mean follow-up of 64 days, the most common adverse events were infections and vomiting, with no deaths or reports of post-lumbar puncture syndrome.

Jinsy Andrews, MD, MSc, FAAN, offered her perspective on the ongoing therapeutic boom in neuromuscular disease and some of the critical needs for physicians to keep up with a rapidly progressing treatment paradigm.

Using qualitative exploratory and quantitative validated questionnaires, investigators saw preparation and assessment time reduced by one-third, with a 60% increase in patient encounters to subspecialty services, compared with the period prior to the COVID-19 pandemic.

The president and CEO of the Muscular Dystrophy Association spoke about the highlights of this year’s annual meeting and the recent therapeutic advances for neuromuscular disorders. [WATCH TIME: 3 minutes]

The chairman of the department of neurology at George Washington University discussed his presentation at MDA 2022 and the value of hearing patient perspective at the conference. [WATCH TIME: 2 minutes]

The director of neuromuscular clinical trials at Columbia University Irving Medical Center shared her perspective on the challenges of familiarizing oneself with the influx of novel medications for neuromuscular diseases. [WATCH TIME: 3 minutes]

Updates from the open-label, delayed-start period of the pivotal 2-part MOXIe trial (NCT02255435) support previous positive primary end points findings from part 2 of the study.

Vamorolone, an investigational agent for Duchenne muscular dystrophy, showed an initial disease-modifying effect that was maintained over a follow-up period of 48 weeks.

The division chief of neuromuscular disorders and vice-chair of research at Virginia Commonwealth University offered his insight into the advances in genetic approaches to LGMD. [WATCH TIME: 2 minutes]

The neurologist and assistant professor at the University of Toronto discussed advantages hyperspectral retinal imaging tools like RetiSpec offer and when clinicians can expect to see them in clinical settings.

The 2022 MDA National Ambassador and patient with Bethlem myopathy spoke to the critical need to improve diagnosis delays in neuromuscular disorders and effective communication between patients and their physicians. [WATCH TIME: 2 minutes]

Data may be useful when analyzing disease progression and treatment efficacy in future facioscapulohumeral muscular dystrophy clinical trials.

The treatment was well tolerated at 48 weeks in patients with DMD with a confirmed mutation amenable to exon 45 skipping.

The staff neurologist and medical director of the Barlo Multiple Sclerosis Program at St Michaels Hospital discussed how investigational BTK inhibitors will be used among an ever-growing MS treatment toolbox. [WATCH TIME: 3 minutes]

Part B of the 2-part study will have patients previously on placebo receive SRP-9001 for an additional 52 weeks.