Latest Conference Coverage

SAGE-718 Demonstrates Safety, Signs of Improvement in Phase 2 Alzheimer Disease Study

Sage Therapeutics’ investigational NMDA receptor positive allosteric modulator was well-tolerated, with signs of cognitive and functional improvements reported for patients with AD in the phase 2 LUMINARY study.

AXS-07 Reduces Migraine Pain in Long-Term Phase 3 MOVEMENT Trial

Through both 24 and 48 hours after single-dose treatment, 85% and 83% of patients, respectively, remained free of the need for rescue medication.

Ganaxolone’s Approval Springboards Future CDD Drug Development: Elia Pestana-Knight, MD

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]

CNM-Au8 Shows Survival Benefit for Patients With ALS in Phase 2 Extension

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.

CNM-Au8 Data From the RESCUE-ALS Open-Label Extension: Matthew Kiernan, MBBS, PhD, DSc, FRACP, FAHMS

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]

What to Expect From the 2022 American Academy of Neurology Annual Meeting

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.

Gaining a Clearer Picture of Gene Therapy in Neuromuscular Disease: Carsten G. Bönnemann, MD

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]

Jinsy Andrews, MD, MSc, FAAN, on Engaging With Patients and Colleagues Amid an Advancing Neuromuscular Field

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 Need for Participation in Neuromuscular Disease Clinical Trials: Donald S. Wood, PhD

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]

BBP-418 Demonstrates Significant Impact on Root Cause of Limb-Girdle Muscular Dystrophy Type 2i

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.

Gene Therapy Zolgensma Improves Bulbar Function in Symptomatic SMA Type 1

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.

Successes and Challenges in the New Era of Genetic Medicine: Nicholas E. Johnson, MD, MSci

The division chief of neuromuscular disorders and vice-chair of research at Virginia Commonwealth University spoke to the ongoing success in the development of gene-mediated therapies and the challenges that come along with treating rare neuromuscular disease. [WATCH TIME: 3 minutes]

CNM-Au8 Shows Prolonged Survival Effect in Amyotrophic Lateral Sclerosis

Zolgensma Reinforces Efficacy in SPR1NT Results of Patients With SMA and 3 Copies of SMN2

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.

Initial Phase 4 RESPOND Results, Omaveloxolone Continues to Perform in FA, Wearable Sensors Safe for FSHD

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

Assessing the Growing Treatment Landscape for Myasthenia Gravis, Next Steps in Optimization: Henry Kaminski, MD

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]

Initial Safety Findings From Phase 4 RESPOND Study of Nusinersen Announced

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.

Understanding the Newest Therapeutic Advances in Neuromuscular Diseases

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.

Quality Improvement Project Facilitates Preparation, Treatment Recommendations for Adolescents at Risk for Sleep Disorders

MDA 2022 Highlights the Therapeutic Advances for Neuromuscular Diseases: Donald S. Wood, PhD

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]

From the Lab to Bedside in Myasthenia Gravis: Henry Kaminski, MD

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]

Managing New Therapies for Neuromuscular Diseases: Jinsy Andrews, MD, MSc

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]

New MOXIe Extension Trial Results Consistent With Persistent Effect of Omaveloxolone in Friedreich Ataxia

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 Demonstrates Continued Safety, Efficacy in DMD Delayed-Start Analysis

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 Revolution in Limb-Girdle Muscular Dystrophy With Gene Therapies: Nicholas E. Johnson, MD, MSci

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]

NeuroVoices: Sharon Cohen, MD, FRCPC, on the Diagnostic Potential of Retinal Imaging for Alzheimer Disease

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.

Diagnostic Delays and the Importance of the Physician-Patient Relationship: Amy Shinneman

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]

Wearable Sensors Feasible, Reliable for Measuring Functional Performance in FSHD

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

Casimersen Increases Exon Skipping, Dystrophin Expression in Patients With DMD Amenable to Exon 45 Skipping

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

Where Tolebrutinib and Other BTK Inhibitors Fit in the MS Treatment Landscape: Jiwon Oh, MD, PhD

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]